Re­struc­tur­ing the pipeline, Eli Lil­ly says a baric­i­tinib come­back in rheuma­toid arthri­tis could take years

Eli Lil­ly was sup­posed to mark this year with the launch of a new block­buster med­i­cine for rheuma­toid arthri­tis — wag­ing a com­mer­cial war with some high-pro­file ri­vals. In­stead, it is now fac­ing a years-long de­lay in or­der to mount a new study aimed at re­solv­ing the FDA’s safe­ty fears for baric­i­tinib. And its ri­vals are gain­ing a big head start in a ma­jor mar­ket.

In the mean­time, Lil­ly’s Q2 re­port in­cludes the news that the phar­ma gi­ant is re­or­ga­niz­ing its pipeline, pri­or­i­tiz­ing 10 drugs in the on­col­o­gy port­fo­lio while plan­ning to out­li­cense the rest of the slate.

Lil­ly $LLY had been close-mouthed about the rea­sons for the stun­ning re­jec­tion of baric­i­tinib in April. But in a new state­ment out ear­ly Tues­day, the phar­ma gi­ant made it clear that reg­u­la­tors had pulled the plug on baric­i­tinib af­ter fret­ting about a se­ries of throm­boem­bol­ic events — blood clots di­ag­nosed as deep ve­nous throm­bo­sis and pul­monary em­bolism — in two of sev­en Phase II and Phase III stud­ies.

Run­ning a new tri­al to get the FDA the new da­ta reg­u­la­tors are de­mand­ing and re­sub­mit­ting the ap­pli­ca­tion will take at least 18 months, says Lil­ly, which is one of the slow­er drug de­vel­op­ers among the largest phar­mas in the in­dus­try. Drug re­views al­so typ­i­cal­ly take 10 months to com­plete. And some an­a­lysts, like Leerink’s Sea­mus Fer­nan­dez, are shov­ing this one down the line by four long years.

Notes Fer­nan­dez: “While these up­dates are dis­ap­point­ing, it is con­sis­tent with our mod­eled US in­tro­duc­tion in 2021.”

In­vestors didn’t like the news. Lil­ly’s shares dropped close to 3% while its part­ner In­cyte saw its price slide 2.5%.

Lil­ly, mean­while, ap­pears ready to make some big changes in the can­cer drug pipeline it’s been build­ing. From its Q2 re­lease:

Us­ing this frame­work, Lil­ly will now fo­cus on sev­en pipeline as­sets for pri­or­i­ty in­ter­nal de­vel­op­ment and three ad­di­tion­al as­sets which are pend­ing da­ta from on­go­ing tri­als. The com­pa­ny has or will seek ex­ter­nal part­ners on the oth­er mol­e­cules in clin­i­cal de­vel­op­ment as ap­pro­pri­ate.

On the ta­ble for ac­quir­ers are these 6 Phase II pro­grams:

  • Galu­nis­ert­ib, a TGF-b in­hibitor in Phase II for he­pa­to­cel­lu­lar car­ci­no­ma.
  • Ralime­tinib, a p38 mi­to­gen-ac­ti­vat­ed pro­tein ki­nase in­hibitor in Phase II for ovar­i­an can­cer.
  • Emi­betuzum­ab, a MET an­ti­body in Phase II for non-small cell lung can­cer.
  • A notch in­hibitor
  • An FGFR in­hibitor
  • A CX­CR4 pep­tide in­hibitor

Lil­ly ex­ecs are clear­ly not hap­py about the baric­i­tinib set­back.

“We dis­agree with the FDA’s con­clu­sions, and be­lieve the ex­ist­ing com­pre­hen­sive clin­i­cal da­ta demon­strate there is a pos­i­tive ben­e­fit/risk pro­file that sup­ports baric­i­tinib’s ap­proval as a new treat­ment op­tion for peo­ple suf­fer­ing from RA in the Unit­ed States,” said Christi Shaw, pres­i­dent of Lil­ly Bio-Med­i­cines. Yes, the com­pa­ny, ad­mit­ted, there was an im­bal­ance in events if you look at the con­trolled stud­ies that com­pared the drug with a place­bo. But if you step back the rate was the same over the course of the full de­vel­op­ment pro­gram.

Eu­ro­pean and Japan­ese reg­u­la­tors, Lil­ly ar­gues, were will­ing to deal with the da­ta by adding a note of cau­tion warn­ing pa­tients at risk for DVT and PE.

Clear­ly, the FDA isn’t ac­cept­ing that ar­gu­ment.

In the mean­time, Re­gen­eron and Sanofi have strut­ted ahead to win a de­layed OK on sar­ilum­ab while Ab­b­Vie works on its own suc­ces­sor to Hu­mi­ra, ABT-494. And Gilead is mov­ing con­fi­dent­ly ahead with a mas­sive Phase III pro­gram for fil­go­tinib.

All of these drugs have been tapped as new ther­a­pies that could earn any­where from $2 bil­lion to $3 bil­lion each. This is not a race that Lil­ly could af­ford to lose.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

CEO Markus Warmuth (Monte Rosa)

Monte Rosa rakes in $96M Se­ries B as it pre­pares 'mol­e­c­u­lar glue' plat­form for IND-en­abling stud­ies

About four months after completing an extension to its Series A, Monte Rosa Therapeutics is putting its next foot forward with another heap of cash.

The Boston-based biotech is back with $96 million in Series B financing with a goal to get its lead program ready for IND-enabling studies by the end of the year. Though Monte Rosa is keeping its specific target a secret for now, the company has been researching how to utilize its protein degradation technology in breast cancer and non-small cell lung cancer, among other areas.

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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David Berry (Flagship)

Flag­ship's next big tech­no­log­i­cal bet? The cloud

Earlier this month, Flagship announced their big bet on the software half the industry is talking about, launching the AI and machine learning startup. Now, they and a couple other investors are gambling $100 million on a software that much of the public generally thinks of as a cool, IT afterthought: cloud computing.

The idea, says founder and Flagship partner David Berry, is one of scale: The sheer magnitude of biological data that you can store on cloud technology is unprecedented. And that size, when leveraged properly, can allow you to ask questions and form insights that are similarly unprecedented.

Jim Roberts and Brian Finrow (Lumen Bioscience)

With a $4M fed­er­al grant, Lu­men jumps in­to the Covid-19 treat­ment race

It’s been less than a month since Lumen Bioscience announced a $16 million Series B to engineer spirulina — a nutrient-packed super food — for diseases like traveler’s diarrhea, norovirus and C. difficile colitis. And now, the biotech has pulled in another $4 million to do the same for Covid-19.

The approach is quite similar to other gastrointestinal targets the company is pursuing, co-founders and Brian Finrow and Jim Roberts said. The Seattle-based company is working on a camelid antibody cocktail to combat GI infection common among Covid-19 patients. In a study published in the American Journal of Gastroenterology, a majority of Covid-19 patients showed GI and respiratory symptoms, and 25% had only GI symptoms.

FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the company.

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