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.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

Yong Dai, Frontera Therapeutics CEO

Scoop: Lit­tle-known Or­biMed-backed biotech clos­es $160M round to start gene ther­a­py tri­al

Frontera Therapeutics, a China and US biotech, has closed a $160 million Series B and received regulatory clearance to test its first gene therapy stateside, Endpoints News has learned.

Led by the largest shareholder, OrbiMed, the biotech has secured $195 million total since its September 2019 founding, according to an email reviewed by Endpoints. The lead AAV gene therapy program is for an undisclosed rare eye disease, according to the source.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Spanish Prime Minister Pédro Sanchez and European Commission President Ursula von der Leyen (AP Photo/Geert Vanden Wijngaert)

EU to launch vac­cine de­vel­op­ment and man­u­fac­tur­ing part­ner­ship with Latin Amer­i­can and Caribbean coun­tries

While European companies, including BioNTech, are focused on increasing vaccine access to African countries by setting up vaccine manufacturing facilities, the European Union is looking westward to Latin America and the Caribbean.

Speaking at a press conference with Spanish Prime Minister Pédro Sanchez, EU Commission president Ursula von der Leyen said that the EU is launching a new initiative for vaccines and medicines manufacturing in Latin America, to get drugs to Latin America and the Caribbean faster.

Matt Kapusta, uniQure CEO

In trou­bled Hunt­ing­ton’s space, uniQure’s gene ther­a­py shows ear­ly promise

In randomized clinical trial data from a small number of patients, Dutch biotech uniQure shared that its gene therapy for Huntington’s disease seems to reduce the amount of the mutant protein responsible for the disease over the course of a year.

In seven patients with early-stage Huntington’s — four who got the treatment and three who got a placebo — mutant huntingtin protein levels in the cerebrospinal fluid decreased by an average of just over 50% in patients who got the gene therapy compared to around a 17% drop in patients who got the placebo after a year.

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DEM BioPharma CEO David Donabedian (L) and executive chair Jan Skvarka

Long­wood sets an­oth­er 'don't eat me' biotech in­to gear with help of for­mer Tril­li­um CEO Jan Skvar­ka

Jonathan Weissman and team are out with a cancer-fighting biotech riding the appetite for those so-called “don’t eat me” and “eat me” signals.

The scientific co-founder — alongside fellow Whitehead Institute colleague Kipp Weiskopf and Stanford biologist Michael Bassik — has launched DEM BioPharma with incubator Longwood Fund and a crop of other investors.

In all, the nascent, 10-employee biotech has $70 million to bankroll hematology- and solid tumor-based programs, including a lead asset that could enter human trials in two to three years, CEO David Donabedian told Endpoints News.

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De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.