Luigi Naldini, Pierluigi Paracchi, Carlo Russo. Genenta

Ital­ian gene ther­a­py play­er draws new in­vestor from Chi­na for its an­swer to CAR-T re­laps­es

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.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

Af­ter 3 deaths rock the field, gene ther­a­py re­searchers con­tem­plate AAV's fu­ture

Nicole Paulk was scrolling through her phone in bed early one morning in June when an email from a colleague jolted her awake. It was an article: Two patients in an Audentes gene therapy trial had died, grinding the study to a halt.

Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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Jackie Fouse, Agios CEO

Agios scores its sec­ond pos­i­tive round of da­ta for its lead pipeline drug — but that won't an­swer the stub­born ques­tions that sur­round this pro­gram

Agios $AGIO bet the farm on its PKR activator drug mitapivat when it recently decided to sell off its pioneering cancer drug Tibsovo and go back to being a development-stage company — for what CEO Jackie Fouse hoped would be a short stretch before they got back into commercialization.

On Tuesday evening, the bellwether biotech flashed more positive topline data — this time from a small group of patients in a single-arm study. And the executive team plans to package this with its earlier positive results from a controlled study to make its case for a quick OK.

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Vir's CMO says he's sur­prised that a low dose of their he­pati­tis B drug ap­pears promis­ing in ear­ly slice of da­ta — shares soar

Initial topline data from a Phase I study of a new therapeutic for chronic hepatitis B virus was so promising that it surprised even the CMO of the company that produces it.

Vir Biotechnology on Tuesday announced that its VIR-3434 molecule reduced the level of virus surface antigens present in a blinded patient cohort after eight days of the trial with just a single 6 mg dose. Six of the eight patients in the cohort were given the molecule, and the other two a placebo—all six who received the molecule saw a mean antigen reduction of 1.3 log10 IU/mL, Vir said.

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Eli Lil­ly demon­strates that 2 an­ti­bod­ies beat 1 for guard­ing against se­vere Covid-19. But can that solve the first an­ti­body’s prob­lem amid slow up­take?

It seems safe to say that two antibodies are better than one.

Eli Lilly released the largest results yet on Tuesday for their Covid-19 neutralizing antibody cocktail, announcing that the combo reduced deaths and hospitalizations in coronavirus patients by 70%. Across 1,000 patients, there were 11 such events in the treatment group and 36 in the placebo group.

The breakdown for deaths alone was even starker: 10 in the placebo group and 0 in the treatment group. Lilly added that the drug hit secondary endpoints for reducing viral load and alleviating symptoms, although they did not disclose numbers.

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George Yancopoulos (L) and Len Schleifer (Regeneron)

Re­gen­eron touts pos­i­tive pre­lim­i­nary im­pact of its Covid an­ti­body cock­tail, pre­vent­ing symp­to­matic in­fec­tions in high-risk group

Regeneron flipped its cards on an interim analysis of the data being collected for its Covid-19 antibody cocktail used as a safeguard against exposure to the virus. And the results are distinctly positive.

The big biotech reported Tuesday morning that their casirivimab and imdevimab combo prevented any symptomatic infections from occurring in a group of 186 people exposed to the virus through a family connection, while the placebo arm saw 8 of 223 people experience symptomatic infection. Symptomatic combined with asymptomatic infections occurred in 23 people among the 223 placebo patients compared to 10 of the 186 subjects in the cocktail arm.

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IPO track­er: 2021 gets start­ed with a flur­ry of new of­fer­ings

A global pandemic couldn’t slow down what turned out to be a record year for biotech IPOs. With the calendar turning toward 2021, the Endpoints News team is prepped to track each new filing this year, and the outcome. We’re off to another hot start at least.

Below, you’ll find the companies that have filed to go public, in addition to those that have already priced. Through the first two business weeks of January, there have already been 9 biotechs that have filed or priced, and the number is only expected to grow. We’ll keep the tracker updated as it does.

Matt Gline (L) and Vivek Ramaswamy

Scoop: Vivek Ra­maswamy is hand­ing the CEO job to a top lieu­tenant at Roivant — but he’s not ex­act­ly leav­ing the biotech scene

Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

He launched his first biotech with an Alzheimer’s drug he picked up cheap, and watched the experiment implode in one of the highest profile pivotal disasters seen in the last decade. But it didn’t slow the 30-something exec down; if anything, he hit the accelerator. Ramaswamy blazed global paths and went on to raise billions to spur the creation of a large lineup of little Vants promising big things at a fast pace. He sold off a section of the Vant brigade to Sumitomo Dainippon for $3 billion. And more recently the relentless dealmaker has been building a computational discovery arm to add an AI-driven approach to kicking up new programs and companies, supplementing the in-licensing drive while pursuing advances that have created more than 700 jobs at Roivant, with $2 billion in reserves.

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