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.

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.