Num­ber 7? Cel­gene part­ner BeiGene joins the crowd with stel­lar PD-1/L1 da­ta from a piv­otal test

Get ready for the sec­ond wave of PD-1/L1 check­point in­hibitors.

BeiGene $BGNE an­nounced just ahead of the start of the week in Bei­jing that their PD-1 tislelizum­ab — part­nered with Cel­gene $CELG — scored stel­lar da­ta in a small Phase II tri­al for clas­si­cal Hodgkin’s lym­phoma among pa­tients who had ei­ther failed or couldn’t take au­tol­o­gous stem cell trans­plan­ta­tion. This is their first piv­otal tri­al, and it un­der­scores why Cel­gene inked a $1.4 bil­lion deal to buy in­to the drug — which may end up as the 7th new check­point to hit the glob­al mar­ket.

Jane Huang

This is a very fa­mil­iar are­na for the check­point field. Bris­tol-My­ers Squibb’s Op­di­vo gained an ac­cel­er­at­ed ap­proval back in 2016 for cHL — with a 7% rate of CRs — and Mer­ck was close be­hind with an OK a year ago as a 4th line ther­a­py fol­low­ing promis­ing re­sults for Keytru­da.

BeiGene, though, would be first in Chi­na, with a sol­id show­ing un­der­scor­ing their drug’s po­ten­tial.

In this new case their drug hit an ob­jec­tive re­sponse rate of 73%, with a 50% CR, and the me­di­an du­ra­tion of re­sponse — over a 6-month av­er­age fol­low-up — had not been reached. 

The transpa­cif­ic BeiGene plans to hus­tle up an ap­pli­ca­tion in Chi­na while they work with Cel­gene on a late-stage pro­gram that in­cludes tri­als for “non-small cell lung can­cer, he­pa­to­cel­lu­lar car­ci­no­ma, and esophageal squa­mous cell car­ci­no­ma; as well as two glob­al Phase 2 tri­als in pa­tients with pre­vi­ous­ly treat­ed he­pa­to­cel­lu­lar car­ci­no­ma or with R/R ma­ture T-and NK-cell lym­phomas, and an ad­di­tion­al piv­otal Phase 2 tri­al in Chi­na in urothe­lial can­cer.”

That’s not all.

BeiGene al­so an­nounced that af­ter dis­cus­sions with the FDA they’re prep­ping an NDA for the first half of 2019 with plans to pur­sue an ac­cel­er­at­ed ap­proval of their drug zanubru­ti­nib for pa­tients with Walden­ström macroglob­u­line­mia based on their glob­al Phase I study. “A fi­nal de­ter­mi­na­tion to sub­mit the NDA will be made sub­se­quent to the pre-NDA meet­ing with FDA af­ter ob­tain­ing ma­ture da­ta from the study this fall.”

Right now there are 5 PD-1/L1 check­points on the mar­ket. Re­gen­eron $REGN and Sanofi $SNY plan to make that 6. And BeiGene looks well po­si­tioned to hit the 7th spot. As of now, there are still hun­dreds of on­go­ing tri­als, in­clud­ing a mob of com­bo stud­ies, as Mer­ck dukes it out with Bris­tol-My­ers for the top po­si­tion of a grow­ing mega­mar­ket. As of now, Mer­ck looks set to take the lead with a string of suc­cess­es in front­line lung can­cer.

Many of these new check­points are be­ing stud­ied in Chi­na, where a group of deep-pock­et in­vestors have been birthing new bio­phar­ma com­pa­nies look­ing to fill the gap of drug tech that sep­a­rates the boom­ing Asian mar­ket with the West.

But there are al­so more than 150 of these ex­per­i­men­tal PD-1/L1 drugs in the glob­al pipeline, ac­cord­ing to re­search from the Can­cer Re­search In­sti­tute, and even­tu­al­ly the world will ei­ther run out of un­chart­ed on­col­o­gy ter­rain, mar­ket po­ten­tial or pa­tients look­ing to en­roll in a tri­al. Then the new ar­rivals will ei­ther shriv­el or the whole mar­ket will be swift­ly com­modi­tized with mul­ti­ple play­ers.

That line, though, has not yet been reached.

“We are ex­cit­ed to an­nounce the pre­lim­i­nary topline re­sults from our first piv­otal tri­al for tislelizum­ab. De­spite short fol­low-up, we be­lieve there was a demon­stra­tion of ro­bust ac­tiv­i­ty, with high over­all and com­plete re­sponse rates in ad­di­tion to a safe­ty pro­file that is con­sis­tent with oth­er PD-1 in­hibitors. We be­lieve these strong re­sults will sup­port our first reg­u­la­to­ry fil­ing in Chi­na for tislelizum­ab, which is planned for lat­er this year,” com­ment­ed Jane Huang, chief med­ical of­fi­cer, hema­tol­ogy, at BeiGene.

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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Hal Barron, Endpoints UKBIO19

GSK, Vir's hopes for a Covid-19 an­ti­body fall flat in NIH 'mas­ter pro­to­col' with no ben­e­fit in hos­pi­tal­ized pa­tients

GlaxoSmithKline and Vir Biotechnology were hopeful that one of their partnered antibodies would carve out a win after getting the invite to a major NIH study in hospitalized Covid-19 patients. But just like Eli Lilly, the pair’s drug couldn’t hit the mark, and now they’ll be left to take a hard look at the game plan.

The NIH has shut down enrollment for GSK and Vir’s antibody VIR-7831 in its late-stage ACTIV-3 trial after the drug showed negligible effect in achieving sustained recovery in hospitalized Covid-19 patients, the partners said Wednesday.

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As Brain­Storm con­tin­ues to tout ‘clear sig­nal’ on ALS drug, the FDA of­fers a rare pub­lic slap­down on the da­ta

A little more than a week after BrainStorm acknowledged that regulators at the FDA had informed them that the biotech needed more data before it could expect to gain an approval for its ALS treatment NurOwn — while still touting a “clear signal” of efficacy and not ruling out an application — the agency has decided to clarify the record in a most unusual statement.

The FDA statement amounts to a straight slap own, offering a different set of efficacy numbers from the company’s public presentation last November and ruling out any chance of statistical significance.

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Eli Lil­ly claims suc­cess in a new JAK in­di­ca­tion: hair loss

Over the last decade, drugmakers have proven JAK inhibitors can treat a smattering of immune-related diseases ranging from rheumatoid arthritis to Covid-19. Now Eli Lilly has pulled out a new one.

Lilly and its biotech partner Incyte announced Wednesday that their JAK inhibitor baricitinib effectively regrew patients’ hair in a Phase III trial for alopecia areata, an autoimmune condition that can cause sudden, severe and patchy hair loss. Lilly didn’t break down the results from the 546-patient trial, but the primary endpoint was improvement on a standard score for alopecia symptoms.

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In­tro­duc­ing End­points FDA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.

Thank you, next: Take­da hands Ovid $196M cash to rein back in Phase III-ready seizure drug, re­viv­ing bat­tered stock

Soticlestat made it.

Takeda is bringing the drug back into its fold more than four years after first entrusting the team at Ovid with the mid-stage clinical work. For all that — generating what they saw as positive Phase II data in Dravet syndrome and Lennox-Gastaut syndrome — the biotech has been rewarded with $196 million in upfront cash, with another $660 million reserved for regulatory and commercial milestones.

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Michael Shpigelmacher

Khosla joins bet on un­con­ven­tion­al start­up look­ing to send drug de­liv­er­ing ro­bots in­to the brain

When Michael Shpigelmacher started the project, he knew he’d have to fund it himself. Every other effort of its kind was academic, rejected as too risky by investors.

Shpigelmacher, a robotics geek and entrepreneur who had drifted into consulting for pharma, wanted to build the real-life equivalent of technology from the 1960s film “Fantastic Voyage,” the one where a submarine crew is shrunk to “about the size of a microbe” and sent on a mission to repair a scientist’s brain. He scanned the literature, found the lab that was working on the most advanced project — at the Max Planck Institute in Germany, it turned out — and started funding them with money from his and his co-founders’ own accounts, along with some seed cash from friends and family.

Antoine Papiernik, Sofinnova managing director (Business Wire)

Sofinno­va Part­ners stays fo­cused on late-stage deals with a new, $540M crossover fund

One of Europe’s most high-profile biopharma investors is getting $540 million to invest in new crossover deals for late-stage companies.

The Paris-based VC says the fresh Sofinnova Crossover Fund raise positions them as the “largest crossover investor in Europe dedicated to late-stage biopharma and medtech investments.”

They got a leg up in France after winning a special “Tibi” designation from the French government, giving them access to a pool of €6 billion that helped them gain an edge with institutional investors. Since they were founded close to 50 years ago, the venture group has backed more than 500 companies and currently has more than €2 billion under management.