Cel­lec­tis preps for ground­break­ing US tri­als for an off-the-shelf CAR-T ther­a­py

Cel­lec­tis is ramp­ing up the first-ever US tri­al launch­es for an off-the-shelf CAR-T ther­a­py.

The Paris-based biotech $CLLS, which has a siz­able R&D group in Man­hat­tan, says the FDA has signed off on their IND for a Phase I study of UCART123 in acute myeloid leukemia and blas­tic plas­ma­cy­toid den­drit­ic cell neo­plasm.

The plan now is to get start­ed in the next few months, wrap up their ear­ly-stage work in ear­ly 2018 and then launch what they hope will be a reg­is­tra­tional Phase II tri­al in mid-2018 — pro­vid­ed the FDA signs off, Dr. Loan Hoang-Sayag, Cel­lec­tis’ chief med­ical of­fi­cer, tells End­points News via email.

Servi­er and part­ner Pfiz­er have been col­lab­o­rat­ing with Cel­lec­tis on an on­go­ing Phase I study for the lead ther­a­py, UCART19, in the UK. UCART123 is a sole­ly-owned Cel­lec­tis pro­gram us­ing TAL­EN en­gi­neered T cells.

The biotech is com­ing in be­hind lead au­tol­o­gous CAR-T play­ers Kite and No­var­tis, both of which are an­gling for the first-ever ap­proval this year for reengi­neered cells ex­tract­ed from pa­tients. Cel­lec­tis, helmed by CEO An­dre Chouli­ka, has been steadi­ly ad­vanc­ing these off-the-shelf ther­a­pies as an eas­i­er to man­u­fac­ture and de­liv­er can­cer treat­ment that could be a nat­ur­al suc­ces­sor to the first CAR-Ts to hit the mar­ket.

It’s a tricky process though. Their ther­a­py has been test­ed in com­pas­sion­ate use cas­es, and in one in­stance re­quired steroids to tamp down on an im­mune re­sponse, which is one re­ac­tion they def­i­nite­ly don’t want to see in an al­lo­gene­ic ap­proach. The au­tol­o­gous lead­ers have been us­ing pa­tient-de­rived cells pre­cise­ly in or­der to avoid any im­mune re­sponse.

UCART123 is a gene-edit­ed T-cell in­ves­ti­ga­tion­al drug that tar­gets CD123, an anti­gen ex­pressed at the sur­face of leukemic cells in AML, tu­moral cells in BPD­CN. The clin­i­cal re­search for AML will be led at Weill Cor­nell by prin­ci­pal in­ves­ti­ga­tor Dr. Gail J. Roboz, sirec­tor of the Clin­i­cal and Trans­la­tion­al Leukemia Pro­grams. The UCART123 clin­i­cal pro­gram for BPD­CN will be led at the MD An­der­son Can­cer Cen­ter by Dr. Naveen Pem­mara­ju, an as­sis­tant pro­fes­sor, and Pro­fes­sor Hagop Kan­tar­jian, de­part­ment chair of the De­part­ment of Leukemia, Di­vi­sion of Can­cer Med­i­cine.

Hoang-Sayag called the move “a ma­jor mile­stone not on­ly for the com­pa­ny but al­so for the med­ical com­mu­ni­ty, glob­al biotech and phar­ma­ceu­ti­cal in­dus­tries at large. Cel­lec­tis’ al­lo­gene­ic UCART prod­ucts have the po­ten­tial to cre­ate an im­por­tant shift with re­gard to avail­abil­i­ty, and cost-ef­fec­tive­ness, to make these ther­a­pies wide­ly ac­ces­si­ble to pa­tient pop­u­la­tion across the world.”

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Bio­gen touts new ev­i­dence from the gene ther­a­py com­pa­ny it wa­gered $800M on

A year ago, Biogen made a big bet on a small gene therapy company. Now they have new evidence one of their therapies could work.

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Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

In fi­nal re­port, ICER ap­pears to have a change of heart on new acute mi­graine ther­a­pies

ICER appears to have reversed course on the fresh crop of acute migraine therapies.

The cost-effectiveness watchdog in November issued a draft report suggesting that existing generic medicines are more effective and cheaper than Allergan’s December-approved CGRP ubrogepant, Biohaven rival molecule, rimegepant (which is under FDA review), and Lilly’s October-sanctioned lasmiditan, which binds to 5-HT1F receptors.

Anthony Fauci (AP Images)

UP­DAT­ED: NIH-part­nered Mod­er­na ships off its PhI-ready coro­n­avirus vac­cine can­di­date to a sea of un­cer­tain­ty

Off it goes.

Moderna has shipped the first batch of its mRNA vaccine against SARS-CoV-2 from its manufacturing facility in Norwood, Massachusetts, to the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland, for a pioneering Phase I study.

It’s a hectic race against time. In the 42 days since Moderna selected the sequence they would use to develop their vaccine — a record time, no less — the number of confirmed cases around the world has surged astronomically from a few dozen to over 80,000, per WHO and Johns Hopkins estimates.

The candidate that they came up with, mRNA-1273, encodes for a prefusion stabilized form of the spike protein, which gives the virus its crown shape and plays a key role in transmission. The Coalition for Epidemic Preparedness Innovations, the Oslo-based group better known as CEPI, funded the manufacture of this batch.

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Bi­cy­cle Ther­a­peu­tics takes Roche's Genen­tech on an up to $2B im­muno-on­col­o­gy ride

Bicycle Therapeutics — which is developing a new class of chemically synthesized drugs designed to be pharmacologically as active as biologics, yet manufactured as small molecules —  has scored another big partner: Roche’s Genentech.

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When drug val­ue as­sess­ment meets re­al-world ev­i­dence: ICER en­lists Ae­tion in pric­ing eval­u­a­tion

In a union of two of the hottest trends in the US biopharma world, ICER is teaming up with a high-profile company to integrate real-world evidence in their assessment of treatment value.

The drug pricing watchdog — formally the Institute for Clinical and Economic Review — said it will utilize Aetion’s evidence platform in “select upcoming assessments” and their new 24-month re-evaluations of drugs granted accelerated approval by the FDA.

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First US Covid-19 tri­als set to get un­der­way in Ne­bras­ka and Wash­ing­ton, backed by NIH

The first US clinical trials on the novel coronavirus are scheduled to get underway next month at the University of Nebraska Medical Center, where American passengers were taken after being evacuated from the Diamond Princess cruise ship, and at the Kaiser Permanente Washington Health Research Institute. Both trials are sponsored by the NIH’s National Institute for Allergy and Infectious Diseases, which has led the US’s medical response to the outbreak.

Mallinck­rodt, once the na­tion’s largest oxy­codone pro­duc­er, an­nounces ten­ta­tive $1.6B set­tle­ment

Three years after it first paid out fines for its role in the US opioid abuse epidemic, Mallinckrodt has announced an agreement-in-principle that will see the company pay out $1.6 billion and place its generics unit in bankruptcy.

The tentative deal would settle hundreds of lawsuits from state and local governments over Mallinckrodt’s role in the epidemic, while also helping address the company’s increasingly mountainous debt. Although Purdue Pharma has drawn the bulk of both public and legal acrimony for opioid sales, documents made public earlier this year showed that Mallinckrodt subsidiary SpecGx, along with the generic subsidiaries of Teva and Endo Pharmaceuticals, accounted for the vast majority of the 76 billion opioid pills distributed from 2006 to 2012. Mallinckrodt was at the top of that list.