Pfiz­er, Mer­ck KGaA score a land­mark FDA OK for check­point con­tender avelum­ab

Lu­ciano Ros­set­ti, Mer­ck Serono

Mer­ck KGaA and Pfiz­er have done it.

The bio­phar­ma part­ners came away with their first FDA ap­proval of the check­point in­hibitor avelum­ab, mak­ing it the fourth drug in this cat­e­go­ry to make it to the mar­ket. They beat As­traZeneca, which will now an­gle for its first ap­proval of dur­val­um­ab as the 5th check­point.

The FDA gave out its ap­proval for Merkel cell car­ci­no­ma un­der an ac­cel­er­at­ed ap­proval process. The ther­a­py, the first for a rare form of skin can­cer, has al­so been giv­en break­through drug sta­tus. The drug is cur­rent­ly in 30 clin­i­cal pro­grams as the two play­ers look to wedge their way in­to a multi­bil­lion-dol­lar mar­ket for drugs that are gain­ing wide use in treat­ing can­cer.

The ap­proval marks an­oth­er big ad­vance for Pfiz­er, which paid $850 mil­lion up­front to part­ner with Ger­many’s Mer­ck — a record sum. The ap­proval of this an­ti-PD-L1 IgG1 mon­o­clon­al an­ti­body al­so marks a ma­jor win for Mer­ck KGaA, which has suf­fered through more than a decade with­out a block­buster OK. Mer­ck KGaA al­so came away with a pack­age of reg­u­la­to­ry and com­mer­cial mile­stones on avelum­ab worth up to $2 bil­lion when it tied up with Pfiz­er in 2014.

The ther­a­py will be sold as Baven­cio. A spokesper­son for Pfiz­er said the drug will cost $13,000 a month whole­sale — ahead of pay­er dis­counts.

The new ap­proval marks just how quick­ly the FDA can move when it wants to. The OK is based on a sin­gle arm study in­clud­ing 88 pa­tients. Of the 88 pa­tients who re­ceived Baven­cio in the tri­al, says the FDA, 33% ex­pe­ri­enced com­plete or par­tial shrink­age of their tu­mors. The re­sponse last­ed for more than six months in 86% of re­spond­ing pa­tients and more than 12 months in 45% of re­spond­ing pa­tients.

Not on­ly was avelum­ab a big suc­cess as a check­point, the de­vel­op­ment pro­gram from start to first ap­proval was ex­e­cut­ed swift­ly.

“Three-and-a-half years af­ter the first-in-hu­man dose es­ca­la­tion stud­ies, we are get­ting our first ap­proval,” says Lu­ciano Ros­set­ti, the head of R&D at Mer­ck Serono. And don’t ex­pect any­one to start sit­ting on lau­rels. Check­point in­hi­bi­tion is a big field, and the Pfiz­er/Mer­ck KGaA team plan to make their mark as the pi­o­neers divvy up lead­er­ship roles in a wide ar­ray of can­cers.

As­traZeneca re­jigged its de­vel­op­ment cam­paign for the next check­point in the pipeline, re­ly­ing heav­i­ly on its work in com­bin­ing it with the CT­LA-4 check­point treme­li­mum­ab to try and leapfrog the fast mov­ing trio in the lead: Mer­ck, Bris­tol-My­ers Squibb and Roche/Genen­tech. They’ll be star­ing down some of the biggest ri­vals in the in­dus­try in a make-or-beak ef­fort to dis­tin­guish its R&D op­er­a­tions, which has had sev­er­al suc­cess­es in can­cer but con­tin­ues to be plagued by set­backs in the clin­ic and at the FDA.

You can add Mer­ck KGaA to the list now.

“You need to have a strong PD1/ PD-L1 to an­chor com­bi­na­tions for oth­er as­sets,” Ros­set­ti tells me. And now they have one ap­proved and en­ter­ing the mar­ket.

The FDA ac­cept­ed their ap­pli­ca­tion for urothe­lial car­ci­no­ma just a few weeks ago, and Ros­set­ti says they’re push­ing hard on ovar­i­an, gas­tric and lung can­cer work that could pave the way for near-term ap­provals.

“To be prac­ti­cal,” he adds, “the biggest op­por­tu­ni­ty is in non-small cell lung can­cer.” One of those big op­por­tu­ni­ties is demon­strat­ing an ef­fect in low PD-L1 ex­pressers with re­searchers al­so ex­plor­ing high in­ten­si­ty ther­a­py for high ex­pressers.

Ros­set­ti, though, is care­ful to of­fer no big promis­es on what the da­ta will show. He’s acute­ly aware that when they mod­i­fied the lung can­cer study, the com­pa­ny was gam­bling on a dis­ease that proved a dis­as­ter for Bris­tol-My­ers Squibb and a chance for Mer­ck to leap ahead of the pack.

The first thing that Ros­set­ti did when he was named R&D chief in 2014 was to fo­cus the pipeline, get­ting rid of 13 pro­grams so they could con­cen­trate on a few se­lect ef­forts like avelum­ab. Now he says the com­pa­ny is in­ter­est­ed in do­ing a few deals to help beef up its fo­cus on com­bi­na­tions and more. But they’ll be very strate­gic about launch­ing new tri­als.

“While skin can­cer is one of the most com­mon can­cers, pa­tients with a rare form called Merkel cell can­cer have not had an ap­proved treat­ment op­tion un­til now,” said Richard Paz­dur, MD, act­ing di­rec­tor of the Of­fice of Hema­tol­ogy and On­col­o­gy Prod­ucts in the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search and di­rec­tor of the FDA’s On­col­o­gy Cen­ter of Ex­cel­lence. “The sci­en­tif­ic com­mu­ni­ty con­tin­ues to make ad­vances tar­get­ing the body’s im­mune sys­tem mech­a­nisms for the treat­ment of var­i­ous types of can­cer. These ad­vance­ments are lead­ing to new ther­a­pies—even in rare forms of can­cer where treat­ment op­tions are lim­it­ed or non-ex­is­tent.”

Con­quer­ing a silent killer: HDV and Eiger Bio­Phar­ma­ceu­ti­cals

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

Am­gen chops 172 more staffers in R&D, op­er­a­tions and sales amid neu­ro­science ex­it, rev­enue down­turn

Neuroscience wasn’t the only unit that’s being hit by a reorganization underway at Amgen. As well as axing 149 employees in its Cambridge office, the company has disclosed that 172 others nationwide, including some from its Thousand Oaks, CA headquarters, are being let go.

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Ahead of strate­gic up­date, new Sanofi CEO mulls op­tions for con­sumer health­care arm — re­ports

Big pharma has made moves to sharpen its focus on developing new medicines, while slow-growing consumer health divisions fall by the wayside. Looks like another large drugmaker is considering a similar move. On Thursday, reports citing sources indicated that Sanofi is reportedly mulling a joint venture, sale, or a public listing of its consumer health arm.

The French group is in discussions for options that could value the division at $30 billion, Bloomberg and Reuters reported, citing sources familiar with the matter.

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The triple crown in biotech: An all-or-noth­ing bet on an FDA ap­proval of 3 drugs over 16 months starts to­day

Bristol-Myers Squibb’s $74 billion Celgene deal closed as expected Wednesday evening. And now a new clock has begun to tick down for Celgene shareholders who came away from the deal with CVRs — contingent value rights — worth $9 or nothing. Those CVRs start trading today as $BMYRT.

The new deadline they have is the end of March 2021, a little more than 16 months from now, when Bristol-Myers will need to gain approvals on 3 late-stage drugs it’s picking up in the buyout: Ozanimod and liso-cel (JCAR017) are due up at the end of 2020, with bb2121 deadlined at the end of Q1 in 2021.

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Genap­sys fi­nal­ly un­veils vaunt­ed se­quencer, but can it dent Il­lu­mi­na?

Hesaam Esfandyarpour holds what looks like a mini-cooler up to the computer screen in his California office.

Esfandyarpour is in his late-30s, with crows feet creeping up against a youthful face. He wears a gray polo and the device in his hand — with its hard plastic-looking shell, blue-and-white pattern, and a white plastic paddle resembling a handle jutting out the front — might contain diced strawberries and peanut-butter sandwiches to meet mom and the kids at a SoCal park. Instead, Esfandyarpour tells me it’s going to change medicine and biopharma research.

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Almost two years after leaving GSK to launch Brii Bio with a whopping $260 million in funding, Zhi Hong is seeing the trans-Pacific infectious disease specialist he set out to build take shape.

“Our pipeline is coming together,” he told Endpoints News, with 12 partnered assets plus some internal programs.

As its latest partner, AN2 Therapeutics, comes into the limelight for the first time with a $12 million seed round, so is Brii’s plans in the antibiotics space. Brii has obtained China rights to AN2’s antibacterial targeting mycobacterium tuberculosis for multi-drug resistant TB, which it says is in the clinical stage.

UP­DAT­ED: Make that 2 ap­proved RNAi drugs at Al­ny­lam af­ter the FDA of­fers a speedy OK on ul­tra-rare dis­ease drug

Seventeen years into the game, Alnylam’s pivot into commercial operations is picking up speed.
The bellwether biotech $ALNY has nabbed their second FDA OK for an RNAi drug, this time for givosiran, the only therapy now approved for acute hepatic porphyria. This second approval came months ahead of the February deadline — even after winning priority review following their ‘breakthrough’ title earlier.
AHP is an extremely rare disease, with some 3,000 patients in Europe and the US, not all diagnosed, and analysts have projected peak revenue of $600 million to $700 million a year. The drug will be sold as Givlaari.

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David Ricks. Eli Lilly

Eli Lil­ly touts $400M man­u­fac­tur­ing ex­pan­sion, 100 new jobs to much fan­fare in In­di­anapo­lis — even though it's been chop­ping staff

Eli Lilly is pouring in $400 million to beef up manufacturing facilities at its home base of Indianapolis. The investment, which was lauded by the city’s mayor, is expected to create 100 new jobs.

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No­var­tis, Bay­er, Long­wood back ge­nomics start­up to speed search for im­munother­a­py tar­gets

Nearly a century passed between the first proto-immunotherapy attempts in cancer — crude and obscure but nonetheless with some scientific basis — and Jim Allison’s first T cell paper. Thirty-plus years flipped between the discovery of CTLA-4 as an off-switch and the approval of Yervoy. Twenty-two rolled between PD-1’s isolation and Opdiva and Keytruda. 

Longwood co-founder Lea Hachigian is betting she can hasten that. It’s a bet on newly established single-cell genomic analysis tech and the ability to crunch endless troves of data at a rate few others can, and investors including Leaps by Bayer and Novartis Venture Fund just put $39 million behind it. They call it Immunitas.