AACR: Lit­tle Apex­i­gen steals the show with their CD40/Op­di­vo cock­tail as AACR gets un­der­way

Now that the pi­o­neer PD-1 check­points have hit the mar­ket and start­ed earn­ing their bil­lions, re­searchers in on­col­o­gy are fo­cused on the next big thing in can­cer R&D: us­ing new com­bi­na­tions to reach tu­mors that re­main stub­born­ly re­sis­tant to ap­proved ther­a­pies.

To that end, you can count Apex­i­gen as the ear­ly win­ner in the AACR meet­ing in At­lanta.

Joyson Karakun­nel

A group of in­ves­ti­ga­tors or­ga­nized by The Park­er In­sti­tute for Can­cer Im­munother­a­py in part­ner­ship with the Can­cer Re­search In­sti­tute con­duct­ed an ear­ly-stage clin­i­cal tri­al that com­bined a pair of chemother­a­pies with Apex­i­gen’s CD40 drug APX005M, with or with­out Bris­tol-My­ers Squibb’s Op­di­vo. In the first part of the Phase I tri­al for ad­vanced pan­cre­at­ic can­cer, they want­ed to see how CD40 — de­signed to spur an im­mune at­tack — worked with a PD-1, which is de­signed to take the brakes off an im­mune sys­tem at­tack.

This is the first tri­al where the in­sti­tute held the IND as the prin­ci­pal spon­sor, says Joyson Karakun­nel, med­ical di­rec­tor for The Park­er In­sti­tute and vice pres­i­dent-clin­i­cal de­vel­op­ment at Ar­cus Bio­sciences, who took some time on Sun­day to ex­plain the work. And they’re par­tic­u­lar­ly hap­py that Park­er was able to or­ga­nize and ex­e­cute an aca­d­e­m­ic study like this in just 18 months, of­fer­ing a new and more ef­fi­cient ap­proach to drug de­vel­op­ment that Sean Park­er had hoped for when he found­ed the in­sti­tute.

Of the 24 pa­tients who were evalu­able by AACR, 20 ex­pe­ri­enced a re­sponse with tu­mor shrink­age. And a few of those pa­tients have re­spons­es that en­dured for more than 12 months. That’s par­tic­u­lar­ly pos­i­tive for pan­cre­at­ic can­cer, which has been re­sis­tant to PD-1 and where dis­ease pro­gres­sion typ­i­cal­ly starts in about 5 months.

“To quote (first au­thor) Mark O’Hara, you do not see those kind of wa­ter­fall plots, so it is ex­treme­ly en­cour­ag­ing,” says Karakun­nel.

Tox­i­c­i­ty, how­ev­er, is a key con­cern. Thir­teen pa­tients — 54% — dis­con­tin­ued the reg­i­men due to an ad­verse event from the cock­tail. Ten of the ad­verse events were se­ri­ous. In­ves­ti­ga­tors fol­lowed up with me to note that 9 of the 13 con­tin­ued on at least one of the drugs and 4 stopped al­to­geth­er.

So why use Apex­i­gen’s drug? There are oth­er CD40s out there.  

Karakun­nel ex­plains that se­nior au­thor Bob Von­der­hei­de was fa­mil­iar with the Apex­i­gen pro­gram and thought it would be a good choice. Odd­ly, though, while the biotech had no trou­ble dis­cussing its $73 mil­lion raise from a group of Chi­nese in­vestors last sum­mer and put out their own re­lease Sun­day, the com­pa­ny turned down an in­ter­view re­quest.

The tri­al is now push­ing ahead to the Phase II por­tion of the tri­al with the high, 0.3 mg dose of Apex­i­gen’s drug. And the pri­vate, ven­ture-backed Apex­i­gen re­searchers plan to roll out more da­ta on APX005M in com­bi­na­tion ther­a­py for pa­tients with metasta­t­ic or un­re­sectable melanoma who have pro­gressed on an­ti-PD-1/PD-L1 ther­a­py. 

A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

Endpoints News writer Nicole DeFeudis has posted a snapshot of all the companies, universities and hospital-based groups now racing through the clinic, ranking them according to their place in the pipeline as well as the latest remarks available on timelines. And we’ll keep this lineup updated right through the end of the year, as the checkered flags start to fall, possibly as early as October.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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Bayer's Marianne De Backer with Endpoints founder John Carroll, Endpoints@JPM20 (Jeff Rumans for Endpoints News)

UP­DAT­ED: Hunt­ing a block­buster, Bay­er forges an $875M-plus M&A deal to ac­quire women’s health biotech

Bayer has dropped $425 million in cash on its latest women’s health bet, bringing a UK biotech and its non-hormonal menopause treatment into the fold.

KaNDy Therapeutics had its roots in GlaxoSmithKline, which spun out several neuroscience drugs into NeRRe Therapeutics back in 2012. Five years later the team created a new biotech to focus solely on NT-814 — which they considered “one of the few true innovations in women’s health in more than two decades.”

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Giovanni Caforio, Bristol Myers Squibb CEO (Christopher Goodney/Bloomberg via Getty Images)

UP­DAT­ED: Bris­tol My­ers Squibb com­mits $300 mil­lion to com­bat racial dis­par­i­ties, but de­clines to re­lease own de­mo­graph­ic da­ta

After the police killing of George Floyd, a flurry of pharma and biotech companies, executives and investors jumped out to make statements, either expressing support for Black Lives Matter and the protests or condemning systemic racism.

Now, a Big Pharma company is publicly putting some teeth behind those statements. This morning, Bristol Myers Squibb announced they would spend $300 million on a broad effort to reduce racial health disparities, and diversify both their clinical trials and their own executive team and workforce.

Martin Shkreli (AP Images)

Mar­tin Shkre­li's in­fa­mous Dara­prim falls off top 20 most ex­pen­sive drugs list

Martin Shkreli incited a national uproar five years ago when he raised the price of Daraprim by a factor of 56 essentially overnight from $13.50 to $750 per pill. Now that the “Pharma Bro’s” high-priced project has received a generic, it no longer places among the most expensive drugs in the world.

GoodRx is back with the latest update of the top 20 most expensive drugs and Daraprim’s exclusion marks the biggest change. The drug had previously ranked seventh on the list’s last iteration, which came in February before the world went into quarantine. Another of Shkreli’s former companies, Retrophin, saw its Chenodal drug place in the top 10 again.

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VBL an­nounces pos­i­tive PhI­II da­ta for ovar­i­an can­cer drug; Gene­Cen­tric part­ners with Janssen on blad­der can­cer

VBL Therapeutics announced that it is on track in a Phase III trial of its drug VB-111 (ofranergene obadenovec) to treat platinum-resistant ovarian cancer in combination with paclitaxel.

After conducting its second interim analysis, the independent Data Safety Monitoring Committee (DSMC) recommended that Israel-based VBL continue full steam ahead. The committee looked at unblinded overall survival rate, which is the primary endpoint of the study, dubbed OVAL. The first analysis evaluated CA-125 response.

FDA ac­cepts pri­or­i­ty re­view for Re­gen­eron's evinacum­ab, which could join Pralu­ent in cho­les­terol mar­ket

Almost a year ago to the day, Regeneron announced solid Phase III testing results for evinacumab, a compound that’s designed to lower LDL cholesterol in patients who need treatment beyond a PCSK9 inhibitor. As that anniversary approaches, the biotech is potentially one step closer to putting it on the market.

The FDA has granted priority review and accepted a BLA for evinacumab as a supplement to other lipid-lowering therapies in individuals with homozygous familial hypercholesterolemia, Regeneron announced Wednesday morning. Regeneron’s target action date is next February 11.