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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Alaa Halawaa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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No­var­tis touts sev­en years of dura­bil­i­ty da­ta for Zol­gens­ma

The same day that Roche touted positive durability and safety data for its spinal muscular atrophy drug Evrysdi, Novartis also made a splash with its multi-million dollar gene therapy for the disease.

Novartis rolled out interim data from two long-term follow-up studies Monday at the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference. In the first study, LT-001, all children in the trial that were treated after showing symptoms of SMA “maintained all previously achieved motor milestones” up to 7.5 years after being dosed. The average time since Zolgensma was given was 6.86 years.

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'Band­ing to­geth­er': 50 fe­male biotech ex­ec­u­tives lay out plans for board di­ver­si­ty, new com­pa­nies and men­tor­ing founders

Earlier this month, during the Silicon Valley Bank meltdown, Angie You recalled the speed with which female biotech CEOs were helping each other connect with bankers, get their wires through and assuage concerns during a financial implosion.

This past weekend, 50 of about 125 women who are part of that Slack group and a broader coalition self-dubbed the Biotech Sisterhood met in person in Cancun for the second rendition of an annual summit connecting female biotech CEOs. The attendance list doubled that of the inaugural gathering in Arizona 12 months ago.

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Rohan Palekar, 89bio CEO

89bio’s PhII da­ta add to quick suc­ces­sion of NASH read­outs as field seeks turn­around

89bio said its drug was better than placebo at lessening fibrosis without worsening nonalcoholic steatohepatitis, or NASH, in two of three dose groups.

The San Francisco biotech said it thinks the Phase IIb data pave the way for a potential Phase III, following in the footsteps of another biotech in its drug class, Akero Therapeutics. To fund a late-stage study, CEO Rohan Palekar told Endpoints News 89bio “would need to raise additional capital,” with the company having about $188 million at the end of last year.

FDA in­di­cates will­ing­ness to ap­prove Bio­gen ALS drug de­spite failed PhI­II study

Ahead of Wednesday’s advisory committee hearing to discuss Biogen’s ALS drug tofersen, the FDA appeared open to approving the drug, newly released briefing documents show.

Citing the need for flexibility in a devastating disease like ALS, regulators signaled a willingness to consider greenlighting tofersen based on its effect on a certain protein associated with ALS despite a failed pivotal trial. The documents come after regulatory flexibility was part of the same rationale the agency expressed when approving an ALS drug last September from Amylyx Pharmaceuticals, indicating the FDA’s openness to approving new treatments for the disease.

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Roche and Lil­ly team up to de­vel­op blood test to de­tect ear­ly signs of Alzheimer's

Eli Lilly is teaming up with Roche to help develop a blood test to detect early signs of Alzheimer’s disease and determine whether a patient should go for further confirmatory testing.

Roche’s Elecsys Amyloid Plasma Panel (EAPP) measures pTau 181 protein assay and APOE E4 assay in human blood plasma – elevations in pTau 181 are present in the early stages of Alzheimer’s, while the presence of APO E4 is the most common genetic risk factor for the disease.

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Francesco Marincola, newly-appointed Sonata Therapeutics CSO

Kite's head of re­search leaves for Flag­ship start­up Sonata

Another leader is departing Kite Pharma, and will to spend the “last part” of his career exploring how cancer evades the immune system.

Kite’s senior VP and global head of cell therapy research Francesco Marincola left the Gilead CAR-T unit last week for Sonata Therapeutics. Flagship last May unveiled the startup, which was pieced together from two fledgling biotechs Inzen and Cygnal Therapeutics. As CSO, Marincola will lead Sonata’s push to reprogram cancer cells to make them more immunogenic.

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Vipin Garg, Altimmune CEO

Al­tim­mune’s shares halved af­ter in­ter­im look at PhII weight loss drug da­ta

Altimmune’s attempt to catch up to Novo Nordisk and Eli Lilly’s GLP-1 drugs hit an investor snag Tuesday after the biotech shared interim Phase II weight loss data.

The Maryland biotech’s pemvidutide is a GLP-1/glucagon dual receptor agonist meant to activate GLP-1 receptors to squash appetite and glucagon to ramp up energy use. The 2.4 mg dose showed a placebo-adjusted weight loss of 9.7% at week 24 of 48, which Jefferies analysts said would be comparable to Novo Nordisk’s semaglutide (Wegovy) and Eli Lilly’s tirzepatide (Mounjaro).

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