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.

Zo­genix plans quick re­turn to the FDA with their spurned ap­pli­ca­tion on Dravet syn­drome drug — shares spike

Zo­genix shares are claw­ing back some of the val­ue they lost 2 months ago af­ter the FDA hit the biotech with a refuse-to-file no­tice on their ex­per­i­men­tal ther­a­py for Dravet syn­drome. 

Com­pa­ny ex­ecs said this morn­ing that they worked out reg­u­la­tors’  is­sues with the ap­pli­ca­tion for Fin­tepla, which cen­tered on a pair of big prob­lems: the ab­sence of non-clin­i­cal stud­ies need­ed to al­low as­sess­ment of the chron­ic ad­min­is­tra­tion of fen­flu­ramine and the in­clu­sion of an in­cor­rect ver­sion of a clin­i­cal dataset. Now they plan to re­sub­mit in Q3 af­ter get­ting off the hook on both scores — which trig­gered a sigh of re­lief among in­vestors.

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

Turned back at the FDA, Im­muno­Gen is ax­ing 220 staffers, sell­ing pro­grams and hun­ker­ing down for a new PhI­II gam­ble

After being stymied by FDA regulators who were unconvinced by ImmunoGen’s $IMGN desperation shot at an accelerated OK based on a secondary endpoint, the struggling biotech is slashing its workforce, shuttering R&D projects and looking for buyers to pick up some of its experimental cancer assets as it goes back into a new Phase III with the lead drug.

We found out last month that the FDA had batted back their case for an accelerated approval of their antibody-drug conjugate mirvetuximab soravtansine, which had earlier failed a Phase III study for ovarian cancer. Now the other shoe is dropping.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

Novavax site in Gaithersburg, MD. Novavax

Beef­ing up its new gene ther­a­py unit, Catal­ent inks $18M deal to snap up No­vavax fa­cil­i­ties

Catal­ent’s hunt for rapid growth on the gene ther­a­py front — ce­ment­ed with a $1.2 bil­lion ac­qui­si­tion of Paragon Bio­sciences two months ago — has led them to the vac­cine mak­ers at No­vavax.

With an $18 mil­lion pay­ment, Paragon is tak­ing over two No­vavax sites in Gaithers­burg, MD, in­clud­ing more than 100 of the em­ploy­ees al­ready work­ing there. That’s in ad­di­tion to a sig­nif­i­cant re­duc­tion in op­er­at­ing costs, says the com­pa­ny, as No­vavax shifts to re­ly on Paragon for GMP ma­te­ri­als in clin­i­cal tri­als and, even­tu­al­ly, com­mer­cial sup­ply of their prod­ucts.

As­traZeneca chal­lenges Roche on front­line SCLC af­ter seiz­ing an in­ter­im win — and Mer­ck may not be far be­hind

The crowded playing field in the PD-1/L1 marketing game is about to get a little more complex.

This morning AstraZeneca reported that its CASPIAN study delivered a hit in an interim readout for their PD-L1 Imfinzi combined with etoposide and platinum-based chemotherapy options for frontline cases of small cell lung cancer, a tough target which has already knocked back Bristol-Myers’ shot in second-line cases. The positive data  — which we won’t see before they roll it out at an upcoming scientific conference — give AstraZeneca excellent odds of a quick vault to challenging Roche’s Tecentriq-chemo combo, approved 3 months ago for frontline SCLC in a landmark advance.

“This is the first trial offering the flexibility of combining immunotherapy with different platinum-based regimens in small cell lung cancer, expanding treatment options,” noted AstraZeneca cancer R&D chief José Baselga in a statement.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

Bridge­Bio takes crown for biggest biotech IPO of 2019, as fel­low uni­corn Adap­tive rais­es of­fer­ing size and price

Bridge­Bio Phar­ma and Adap­tive Biotech­nolo­gies have not just up­sized IPO of­fer­ings — the pair of uni­corns have al­so raised their of­fer­ing prices above the range, haul­ing in a com­bined $648.5 mil­lion.

Neil Ku­mar’s Bridge­Bio Phar­ma, found­ed in 2015, has a sta­ble of com­pa­nies fo­cused on dis­eases that are dri­ven by de­fects in a sin­gle gene — en­com­pass­ing der­ma­tol­ogy, car­di­ol­o­gy, neu­rol­o­gy, en­docrinol­o­gy, re­nal dis­ease, and oph­thal­mol­o­gy — and can­cers with clear ge­net­ic dri­vers. The start­up mill birthed a pletho­ra of firms such as Ei­dos, Navire, QED Ther­a­peu­tics and Pelle­Pharm, which func­tion as its sub­sidiaries.

Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

We’ve been wait­ing for the ar­rival of Bridge­Bio’s IPO to top off the wave of new biotech of­fer­ings sweep­ing through Nas­daq at the end of H1. And now we learn that it’s been sub­stan­tial­ly up­sized.

Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.

No­var­tis holds back the copy­cat brigade's at­tack on its top drug fran­chise — for now

A fed­er­al judge has put a gener­ic chal­lenge to No­var­tis’ block­buster mul­ti­ple scle­ro­sis drug Gilenya on hold while a patent fight plays out in court.

Judge Leonard P. Stark is­sued a tem­po­rary in­junc­tion ear­li­er this week, forc­ing My­lan, Dr. Red­dy’s Lab­o­ra­to­ries and Au­robindo Phar­ma to shelve their launch plans to al­low the patent fight to pro­ceed. He ruled that al­low­ing the gener­ics in­to the mar­ket now would per­ma­nent­ly slash the price for No­var­tis, even if it pre­vails. 

Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.