So you’ve been treat­ed with one of the new-age can­cer ther­a­pies. What do you do if there’s a re­lapse and the tu­mors re­turns? 

Genen­ta Sci­ence launched four years ago out of Mi­lan in part to an­swer that ques­tion, and to­day they re­ceived $14.4 mil­lion in round three fund­ing that will pro­pel their unique gene ther­a­py through Phase I/II tri­als for mul­ti­ple myelo­ma and glioblas­toma. Backed now by mon­ey from the Chi­nese firm Qianzhan In­vest­ment Man­age­ment and Fidim, the for­mer own­er of the bio­phar­ma­ceu­ti­cal Rot­tapharm, the com­pa­ny is al­so ex­plor­ing ways of de­liv­er­ing gene ther­a­py to sol­id tu­mors that have been hard­er to reach. 

“The prob­lem Genen­ta faces is that you don’t need to be No­bel Prize brain to un­der­stand CAR-T,” CMO Car­lo Rus­so told End­points News, re­fer­ring to the pop­u­lar can­cer cell ther­a­py. “It is in­tu­itive­ly sim­ple. What we are try­ing to do is not.”

CAR-T treat­ments have been hailed in re­cent years for the break­throughs they’ve of­fered in hard-to-treat blood can­cers, but ini­tial en­thu­si­asm has been tem­pered by 1-year re­lapse rates as high as 40%.

The idea of the Genen­ta ther­a­py, called Tem­fer­on, is to pre­vent those re­laps­es by re­build­ing the pa­tient’s im­mune sys­tem with­in the tu­mor sites them­selves, called TEMS. The treat­ment in­volves in­ject­ing a lentivirus for a gene trans­fer in hematopoi­et­ic stem and prog­en­i­tor cells, trig­ger­ing in­ter­fer­on-αex­pres­sion in the tar­get­ed ar­eas. The pa­tient would then be able to fight off fu­ture re­laps­es.

This ther­a­py has ex­cit­ing po­ten­tial for long term care, Rus­so said, but not nec­es­sar­i­ly for tack­ling a fast-act­ing can­cer. He said he could en­vi­sion a fu­ture where front­line rem­e­dy such as CAR-T or chemother­a­py is used to wipe out tu­mors ear­ly and then Tem­fer­on is ad­min­is­tered for long term pre­ven­tion. 

“It’s like a vac­cine,” Rus­so says, “You’re ex­posed to an anti­gen so you will be pre­pared when you are nat­u­ral­ly ex­posed to the anti­gen.”

The mul­ti­ple myelo­ma and glioblas­toma tri­als have al­ready be­gun, Genen­ta CEO Pier­lui­gi Parac­chi told End­points, and the fund­ing will al­low them to ex­pand to 21 and 9 pa­tients in each. Parac­chi said they may al­so look in­to us­ing parts of the funds to open a clin­i­cal tri­al in the Unit­ed States.  

Genen­ta’s pre­clin­i­cal work, led by Lui­gi Nal­di­ni, ex­am­ined Tem­fer­on on an ar­ray of can­cers; mul­ti­ple myelo­ma was cho­sen for the first clin­i­cal tri­al in large part be­cause bone mar­row trans­plants are al­ready part of the dis­ease’s stan­dard of care, re­duc­ing the risk of the ex­per­i­men­tal ther­a­py. 

Like CAR-T, Tem­fer­on works by re­mov­ing bone mar­row cells, treat­ing them with a gene ther­a­py and then rein­ject­ing them in­to the pa­tient. But Genen­ta method us­es in­ter­fer­on, an old can­cer treat­ment that has been all but dis­con­tin­ued be­cause of its risks. 

“The re­ac­tion I get when I talk about in­ter­fer­on is ‘oh my god this is such an old, bor­ing drug,’” Rus­so said.

But be­cause the ther­a­py lim­its the in­ter­fer­on pro­teins to the mi­cro-en­vi­ron­ment around the tu­mor, it can be ef­fec­tive and low-risk, Rus­so said. 

Among oth­er ben­e­fits, the study on glioblas­toma will al­low re­searchers to see in the very near fu­ture if the treat­ment is hav­ing any ef­fect, as this form of brain can­cer is gen­er­al­ly fa­tal with­in 16 to 20 months.

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA.