FDA OKs Tesaro’s ni­ra­parib for ovar­i­an can­cer, hand­ing the biotech a big win on the la­bel

The FDA has wast­ed no time in giv­ing Tesaro $TSRO a broad ap­proval for the use of its PARP drug ni­ra­parib, which will now be mar­ket­ed as Ze­ju­la as a main­te­nance ther­a­py for re­cur­rent ovar­i­an can­cer.

The OK and the la­bel that came with it rep­re­sents a set­back for Myr­i­ad Ge­net­ics $MYGN, which had ar­gued that its com­pan­ion di­ag­nos­tic would be need­ed to iden­ti­fy a spe­cif­ic group of ovar­i­an can­cer pa­tients with a bio­mark­er in­di­cat­ing that they would most like­ly ben­e­fit. But reg­u­la­tors blew past any di­ag­nos­tic qual­i­fi­ca­tion in giv­ing Tesaro the la­bel that it was look­ing for, vin­di­cat­ing CEO Lon­nie Moul­der’s vow that he could get an OK aimed at a large seg­ment of the mar­ket.

Tesaro had ar­gued that the ef­fi­ca­cy da­ta for ni­ra­parib qual­i­fied for an ap­proval for use in pa­tients who are ei­ther HRD pos­i­tive or HRD neg­a­tive. But they were op­posed by a part­ner who had a sol­id busi­ness case for ar­gu­ing that the drug should be re­served for pa­tients who would be most like­ly to ben­e­fit.

In the end, though, Tesaro is left with a big boast in its fa­vor: “Ze­ju­la is the on­ly PARP in­hibitor that has demon­strat­ed a clin­i­cal­ly mean­ing­ful in­crease in pro­gres­sion-free sur­vival (PFS) in women with re­cur­rent ovar­i­an can­cer, re­gard­less of BR­CA mu­ta­tion or bio­mark­er sta­tus.”

The OK al­so helps po­si­tion Tesaro against As­traZeneca, which just de­liv­ered stel­lar Phase III da­ta on its PARP Lyn­parza. Clo­vis, mean­while, gained an ap­proval for its PARP Rubra­ca late last year as a treat­ment for BR­CA-mu­tat­ed ovar­i­an can­cer that had proved re­sis­tant to at least two pri­or ther­a­pies.

It’s a trans­for­ma­tion­al mo­ment for Tesaro. Its drug was list­ed by Eval­u­atePhar­ma as one of the top drugs in the in­dus­try’s pipeline this year, with peak sales ex­pec­ta­tions hov­er­ing close to $2 bil­lion a year. And its stock climbed about 6% in af­ter-mar­ket trad­ing.

The ap­proval came months ahead of the FDA’s PDU­FA dead­line in June, in­di­cat­ing its in­ter­est and un­der­stand­ing of the field.

Mary Lynne Hed­ley, Tesaro

Ze­ju­la’s la­bel notes that it is in­di­cat­ed for the main­te­nance treat­ment of adult pa­tients with re­cur­rent ep­ithe­lial ovar­i­an, fal­lop­i­an tube, or pri­ma­ry peri­toneal can­cer who are in a com­plete or par­tial re­sponse to plat­inum-based chemother­a­py. And Tesaro im­me­di­ate­ly fol­lowed the news of the OK with plans to open up on the R&D pro­gram for the drug.

“Based on the un­prece­dent­ed re­sults of the NO­VA tri­al in women with re­cur­rent ovar­i­an can­cer, we pre­vi­ous­ly an­nounced the ex­pan­sion and re­fine­ment of our PRI­MA and QUADRA tri­als to in­clude a broad pa­tient pop­u­la­tion, and in the case of PRI­MA, elim­i­nat­ed the en­roll­ment re­quire­ment for a bio­mark­er se­lect­ed tu­mor. With the ap­proval of ZE­JU­LA in hand, we will now be­gin to ex­e­cute on our plans to pur­sue po­ten­tial­ly trans­for­ma­tion­al ap­pli­ca­tions of ni­ra­parib in a broad range of metasta­t­ic can­cer in­di­ca­tions,” said Mary Lynne Hed­ley, pres­i­dent and COO of TESARO. “We plan to ex­pand our first-line ovar­i­an can­cer strat­e­gy to in­clude a com­bi­na­tion study that as­sess­es the po­ten­tial ben­e­fit of ni­ra­parib plus an an­ti-PD-1 an­ti­body in the main­te­nance set­ting and ini­ti­ate a clin­i­cal study of ni­ra­parib in com­bi­na­tion with be­va­cizum­ab in pa­tients with a first re­cur­rence of ovar­i­an can­cer, with an in­tent to re­place chemother­a­py in this set­ting. We re­main strong­ly com­mit­ted to study­ing ni­ra­parib in the breast can­cer set­ting and al­so ex­pect to ini­ti­ate a new tri­al of ni­ra­parib in com­bi­na­tion with an an­ti-PD-1 an­ti­body in women with metasta­t­ic triple-neg­a­tive breast can­cer. Fi­nal­ly, our goal to move ni­ra­parib in­to in­di­ca­tions be­yond ovar­i­an and breast can­cers en­com­pass­es plans to ini­ti­ate a reg­is­tra­tion strat­e­gy for the first-line treat­ment of pa­tients with metasta­t­ic non-small cell lung can­cer that in­cludes a phase 2 tri­al of ni­ra­parib in com­bi­na­tion with an an­ti-PD-1 an­ti­body in pa­tients, re­gard­less of PDL-1 tu­mor ex­pres­sion, and a phase 3 tri­al of ni­ra­parib in com­bi­na­tion with an an­ti-PD-1 an­ti­body in pa­tients with high lev­els of PDL-1 tu­mor ex­pres­sion.”

The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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UP­DAT­ED: Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.

A new num­ber 1 drug? Keytru­da tapped to top the 10 biggest block­busters on the world stage by 2024

Analysts may be fretting about Keytruda’s longterm prospects as a host of rival therapies elbow their way to the market. But the folks at Evaluate Pharma are confident that last year’s $7 billion earner is headed for glory, tapping it to beat out the current #1 therapy Humira as AbbVie watches that franchise swoon over the next 5 years.

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John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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Step­ping on Roche's toes, Mer­ck cuts in­to SCLC niche with third-line Keytru­da OK

In the in­creas­ing­ly crowd­ed check­point race, small cell lung can­cer has been a rare area where Roche, a sec­ond run­ner-up, has a lead over the en­trenched lead­ers Mer­ck and Bris­tol-My­ers Squibb. But Mer­ck is fi­nal­ly mak­ing some head­way in that di­rec­tion with the lat­est ap­proval for its PD-1 star.

The lat­est green light en­dors­es Keytru­da in the third-line treat­ment of metasta­t­ic SCLC, where it would be giv­en to pa­tients whose dis­ease ei­ther don’t re­spond to or re­lapse af­ter chemother­a­py, which would have fol­lowed at least one pri­or line of ther­a­py.

Arc­turus ex­pands col­lab­o­ra­tion, adding $30M cash; Ku­ra shoots for $100M raise

→  Rare dis­ease play­er Ul­tragenyx $RARE is ex­pand­ing its al­liance with Arc­turus $ARCT, pay­ing $24 mil­lion for eq­ui­ty and an­oth­er $6 mil­lion in an up­front as the two part­ners ex­pand their col­lab­o­ra­tion to in­clude up to 12 tar­gets. “This ex­pand­ed col­lab­o­ra­tion fur­ther so­lid­i­fies our mR­NA plat­form by adding ad­di­tion­al tar­gets and ex­pand­ing our abil­i­ty to po­ten­tial­ly treat more dis­eases,” said Emil Kakkis, the CEO at Ul­tragenyx. “We are pleased with the progress of our on­go­ing col­lab­o­ra­tion. Our most ad­vanced mR­NA pro­gram, UX053 for the treat­ment of Glyco­gen Stor­age Dis­ease Type III, is ex­pect­ed to move in­to the clin­ic next year, and we look for­ward to fur­ther build­ing up­on the ini­tial suc­cess of this part­ner­ship.”

Neil Woodford. Woodford Investment Management via YouTube

Wood­ford braces po­lit­i­cal storm as UK fi­nan­cial reg­u­la­tors scru­ti­nize fund sus­pen­sion

The shock of Neil Wood­ford’s de­ci­sion to block with­drawals for his flag­ship fund is still rip­pling through the rest of his port­fo­lio — and be­yond. Un­der po­lit­i­cal pres­sure, UK fi­nan­cial reg­u­la­tors are now tak­ing a hard look while in­vestors con­tin­ue to flee.

In a re­sponse let­ter to an MP, the Fi­nan­cial Con­duct Au­thor­i­ty re­vealed that it’s opened an in­ves­ti­ga­tion in­to the sus­pen­sion fol­low­ing months of en­gage­ment with Link Fund So­lu­tions, which tech­ni­cal­ly del­e­gat­ed Wood­ford’s firm to man­age its funds.