Penn team spot­lights a pi­lot ovar­i­an can­cer tri­al and sur­vival rates for a per­son­al­ized can­cer vac­cine

A group of in­ves­ti­ga­tors at Penn are hold­ing out hope that a new type of per­son­al­ized tu­mor vac­cine may break the crip­pling run of fail­ures that blight­ed the first wave of can­cer vac­cines.

In a tri­al among 25 ad­vanced ovar­i­an can­cer pa­tients, re­searchers took au­tol­o­gous den­drit­ic cells — a type of mes­sen­ger cells that present anti­gen ma­te­r­i­al to T cells — and pulsed them to whole-tu­mor cell lysate al­so from the pa­tients. The pa­tients then re­ceived a dose of these tu­mor-ex­posed den­drit­ic cells every three weeks, up to six months. Al­most half of the pa­tients who could be eval­u­at­ed showed a good re­sponse to the vac­cine, as in­di­cat­ed by a big in­crease in the num­ber of T cells specif­i­cal­ly re­ac­tive to tu­mor ma­te­r­i­al. The dif­fer­ence in 2-year over­all sur­vival rates be­tween the “re­spon­der” pa­tients and “non-re­spon­ders”: 100% ver­sus 25%.

With this ap­proach, the team from Uni­ver­si­ty of Penn­syl­va­nia and the Lau­sanne branch of the Lud­wig In­sti­tute for Can­cer Re­search says the vac­cine is primed with the unique set of mu­ta­tions in an in­di­vid­ual tu­mor. Giv­en its ex­po­sure to the whole tu­mor, it can stim­u­late im­mune re­sponse against not just one but “hun­dreds or thou­sands” of tu­mor-as­so­ci­at­ed tar­gets. It is al­so tu­mor-spe­cif­ic, mean­ing the vac­cine-in­duced T cells would be less like­ly to at­tack healthy cells.

To be sure, the study was de­signed pri­mar­i­ly to as­sess safe­ty and fea­si­bil­i­ty. But see­ing that a pa­tient who start­ed with stage 4 ovar­i­an can­cer (and five pri­or cours­es of chemother­a­py) re­mained dis­ease-free for five years af­ter two years of dos­ing, the in­ves­ti­ga­tors think it can prove an ef­fec­tive ap­proach to fight­ing can­cer, es­pe­cial­ly when paired up with oth­er im­munother­a­pies.

“This vac­cine ap­pears to be safe for pa­tients, and elic­its a broad an­ti-tu­mor im­mu­ni­ty — we think it war­rants fur­ther test­ing in larg­er clin­i­cal tri­als,” said lead au­thor Janos Tanyi in a state­ment about the study, pub­lished in Sci­ence Trans­la­tion­al Med­i­cine.

Biotech af­ter biotech has bit­ten the dust in pur­suit of can­cer vac­cines us­ing shared anti­gens, from Bavar­i­an Nordic and Aduro to Sel­l­as and Ar­gos, which flopped bad­ly de­spite de­ploy­ing a per­son­al­ized ap­proach.

Yet that hasn’t stopped de­vel­op­ers from try­ing.

Mod­er­na and BioN­Tech are the bet­ter known play­ers in the sec­ond wave of can­cer vac­cines, with enor­mous piles of cash for their mR­NA pro­grams. As of De­cem­ber 2017, the Can­cer Re­search In­sti­tute count­ed 344 hu­man stud­ies in progress for can­cer vac­cines.

Pa­tient seg­men­ta­tion, man­u­fac­tur­ing and cost will like­ly re­main key hur­dles for suc­cess in this field. But this kind of ear­ly re­sults is ex­act­ly what keeps com­pa­nies hope­ful.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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Joshua Liang, Clover Biopharmaceuticals CEO

With world still in sore need of dos­es, Clover says its Covid-19 vac­cine is 67% ef­fec­tive in Phase III

With concerns about the Delta variant rising and much of the world still in desperate need of vaccine doses, a Chinese biotech announced Wednesday that a new shot has shown positive results in a large trial against Covid-19, including new variants.

Clover Biopharmaceuticals announced Wednesday that its vaccine candidate showed 79% efficacy against the Delta variant in a Phase II/III trial dubbed Spectra, and 67% effective against Covid-19 overall.

Jean Bennett (Brent N. Clarke/Invision/AP Images)

Lux­tur­na in­ven­tor Jean Ben­nett starts a new gene ther­a­py com­pa­ny to tack­le rare dis­eases left be­hind by phar­ma, VCs

A few years ago Jean Bennett found herself in a surprising place for a woman who invented the first gene therapy ever approved in the United States: No one, it seemed, wanted her work.

Bennett, who designed and co-developed Luxturna, approved in 2018 for a rare form of blindness, had kept building new gene therapies for eye diseases at her University of Pennsylvania lab. But although the results in animals looked promising, pharma companies and investors kept turning down the pedigreed ophthalmology professor.

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David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.