Re­gen­eron makes a block­buster case for its PhI­II drug pipeline

While Bio­gen is strug­gling to get its pipeline to per­form, Re­gen­eron ex­ecs put on a demon­stra­tion to­day on how a biotech com­pa­ny with a sec­ond-tier R&D bud­get can per­form bet­ter on the late-stage front than much big­ger com­pa­nies spend­ing much more on re­search. And it led off with bull­ish re­marks on a pair of would-be block­busters, in­clud­ing their top Phase III drug dupilum­ab.

Dur­ing their sec­ond quar­ter re­view, the ex­ec­u­tive team not­ed:

— Sar­ilum­ab is up for an Oc­to­ber 30 PDU­FA date at the FDA, with a re­cent Eu­ro­pean fil­ing at the EMA to fol­low up on. Peak sales es­ti­mates top $1 bil­lion for this drug, which beat megablock­buster Hu­mi­ra in a head-to-head study on rheuma­toid arthri­tis. This will like­ly be the sec­ond drug part­nered with Sanofi to hit the mar­ket – the first was the PC­SK9 drug Pralu­ent, which has had a slow up­take since its in­tro­duc­tion.

— It’s dupilum­ab, though, that is get­ting the full red car­pet treat­ment at Re­gen­eron. The IL-4/IL-13 block­ing drug – al­so part­nered with Sanofi – is ex­pect­ed to get ap­proved in the first half of 2017. Most an­a­lysts note that with stel­lar Phase III da­ta from a slate of atopic der­mati­tis stud­ies, along with the FDA’s break­through drug des­ig­na­tion to help speed things along, that ap­proval is like­ly to land ear­ly. Peak sales es­ti­mates tend to hov­er around $2.5 bil­lion a year, mak­ing it a con­tender to be­come a top 2017 drug. Some op­ti­mistic sell-siders have pushed their peak es­ti­mates to $4 bil­lion or $5 bil­lion.

R&D chief George Yan­copou­los

“Dupilum­ab is re­al­ly a fran­chise un­to it­self,” says R&D chief George Yan­copou­los in the Q2 call with an­a­lysts to­day. The pos­i­tive da­ta in atopic der­mati­tis has spurred their be­lief that the drug has “huge” po­ten­tial. There’s al­ready sig­nif­i­cant pent­up de­mand from pa­tients who ei­ther don’t re­spond to cur­rent­ly avail­able reme­dies or can’t tol­er­ate them, says Re­gen­eron’s ex­ec­u­tive team. There’s al­so no oth­er com­pe­ti­tion in the late-stage pipeline to con­tend with.

“This re­al­ly has a chance to pro­vide some­thing to pa­tients that don’t have any oth­er al­ter­na­tives at this point,” says Yan­copou­los. And the com­pa­ny is push­ing ahead in asth­ma and oth­er in­di­ca­tions, look­ing to push the bound­aries on its fran­chise po­ten­tial.

— Then there’s REGN 2810, a PD-1 check­point drug al­so part­nered with Sanofi which is al­ready in a pos­si­ble piv­otal study. This is part of an­oth­er am­bi­tious col­lab­o­ra­tion deal with Sanofi, which is look­ing to fi­nal­ly get things go­ing in the can­cer are­na.

Re­gen­eron al­so has a brand new al­liance of its own with Adicet and its CEO, Aya Jakobovits, who earned a big en­dorse­ment from Yan­copou­los.

“We re­al­ly be­lieve in peo­ple and we be­lieve in syn­er­gies,” says the R&D chief, tout­ing a match-up on Adicet’s off-the-shelf ap­proach to de­vel­op­ing cell ther­a­pies. “We have had a long­stand­ing in­ter­est to work with Aya Jakobovits. We have enor­mous re­spect for her and her ca­pa­bil­i­ties.”

Be­yond that, though, Yan­capou­los wasn’t say­ing much about the tech­nol­o­gy and what dis­tin­guish­es it from ri­vals in the field.

— And fi­nal­ly there’s fas­inum­ab (REGN475), an an­ti-NGF pain drug for os­teoarthri­tis, which is up against some com­pe­ti­tion af­ter Pfiz­er and Eli Lil­ly got things go­ing again af­ter a long pause to as­sess safe­ty is­sues. Pa­tients had a bad habit of blow­ing out their joints af­ter tak­ing the drug, and de­vel­op­ers had to be care­ful to ex­clude pa­tients at risk.

Put it all to­geth­er, and Re­gen­eron’s rel­a­tive­ly mod­est $1.5 bil­lion R&D bud­get – aug­ment­ed by Sanofi – is out­per­form­ing most of its peers, and many much larg­er com­pa­nies that have yet to nail down a suc­cess­ful R&D strat­e­gy.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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UP­DAT­ED: Pfiz­er hits the brakes on their piv­otal tri­al for a BC­MA/CD3 bis­pe­cif­ic on safe­ty con­cerns while FDA road­block is hold­ing up Duchenne MD PhI­II

Pfizer’s ambitious plan to take a Phase II study of its BCMA CD3-targeted bispecific antibody elranatamab (PF-06863135) and run it through to an accelerated approval has derailed.

The pharma giant said in a release this morning that they have halted enrollment for their MagnetisMM-3 study after researchers tracked three cases of peripheral neuropathy in the ongoing Phase I. They are now sharing info with the FDA as they explore the red safety flag.

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Stéphane Bancel at the Endpoints #JPM20 breakfast panel in San Francisco, January 2020 (Photo: Jeff Rumans, Endpoints News)

Mod­er­na says Covid-19 vac­cine boost­er in­creased im­mune re­sponse against vari­ants of con­cern

About a month after announcing their variant-specific Covid-19 vaccine boosters showed promising results in mice, Moderna says it now has some human data to back it up.

Volunteers given a booster shot about six to eight months after receiving their second dose saw increased antibody levels against SARS-CoV-2 and two variants of concern: B.1.351, which was first identified in South Africa, and P.1, first identified in Brazil, the company said on Wednesday.

FDA ex­tends re­search agree­ment with MIT-li­censed or­gan-on-chip sys­tems

The FDA on Wednesday extended its four-year agreement with CN Bio, a developer of single- and multi-organ-on-chip systems used for drug discovery, for another three years.

CN Bio said the scope of the research performed by the FDA’s Center for Drug Evaluation and Research has expanded to include the exploration of the company’s lung-on-a-chip system to help with the agency’s evaluation of inhaled drugs, in addition to the agency’s work on its liver model.

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In quest to meet user fee goals, FDA’s per­for­mance con­tin­ues down­ward trend

A recent update to the FDA’s running tally of how it’s meeting its user fee-related performance goals during the pandemic shows an agency that is not out of the woods yet.

The latest numbers reveal that for a second straight quarter in 2021, the FDA has met its user fee goal dates for 93% of original new drug applications, which compares with 94% and 98% for the previous two quarters in 2020, respectively.

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Cynthia Butitta (L) and Joe Jimenez

Is that an­oth­er IPO in the mak­ing? Ex-No­var­tis CEO Joe Jimenez and a lead Kite play­er take up new posts at an off-the-shelf ri­val to 2 pi­o­neer­ing drugs

Right on the heels of taking on a $160 million crossover round in a likely leap to Nasdaq, Century Therapeutics CEO Lalo Flores is now pushing ahead with the high-profile ex-Novartis chief Joe Jimenez as chairman.

Jimenez’s greatest fame at Novartis was earned for one of its weakest products, as their pioneering personalized CAR-T Kymriah won the honors for the first such drug to make it to the market. Now a host of players, including Century, are barreling in behind the frontrunners with allogeneic rivals that can be created for off-the-shelf use.

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Joe Wiley, Amryt CEO

A biotech with a yen for pricey rare dis­ease drugs — and bar­gain base­ment shop­ping — adopts an­oth­er or­phan in lat­est M&A pact

After making it through a long, painful haul to get past a CRL and on to an FDA approval last summer, little Chiasma has found a buyer.

Amryt $AMYT, a company known for its appetite for acquiring expensive drugs for rare diseases at bargain prices, snagged Chiasma and its acromegaly drug Mycapssa (octreotide) capsules in an all-stock deal — with an exchange of 0.396 shares of Amryt for every share of Chiasma.

Ngozi Okonjo-Iweala, Director general of WTO (AP Photo/Keystone/Alessandro Della Bella)

Opin­ion: Waiv­ing Covid-19 vac­cine IP could save lives, but where is the man­u­fac­tur­ing ca­pac­i­ty?

Droves of House Democrats in Washington and members of the European Parliament have now glommed onto a major push by India and South Africa at the WTO to abolish all IP around Covid-19 vaccines.

At first blush, waiving this IP sounds like an easy win: More Covid-19 vaccines made locally for more people means more lives saved. Simple enough, especially as low-income countries have received just a tiny fraction of the world’s vaccine allotment so far.