Fol­low­ing EMA, FDA re­stricts use of high-dose Xel­janz, warn­ing of added risks for the block­buster JAK

The FDA has come out with new re­stric­tions on the use of Pfiz­er’s high-dose ver­sion of Xel­janz af­ter re­searchers drew a di­rect line be­tween the 10 mg dose and a high­er risk of blood clots and death.

In a new safe­ty warn­ing out to­day, the agency added a boxed warn­ing for the block­buster JAK in­hibitor, ad­vis­ing physi­cians of the risk and or­der­ing them to re­strict the ap­proved 10 mg dose for ul­cer­a­tive col­i­tis to “cer­tain pa­tients who are not treat­ed ef­fec­tive­ly or who ex­pe­ri­ence se­vere side ef­fects with cer­tain oth­er med­i­cines.”

Though it wasn’t ap­proved for rheuma­toid arthri­tis, re­searchers had been us­ing the 10 mg dose in a study for RA when they spot­ted the dan­ger­ous im­bal­ance in out­comes, which alarmed reg­u­la­tors on both sides of the At­lantic. In Eu­rope, the 10 mg dose is now banned among pa­tients with heart fail­ure, can­cer, as well as an iden­ti­fi­able risk of de­vel­op­ing blood clots.

The new re­stric­tions fol­low the 2018 OK on UC, its third in­di­ca­tion fol­low­ing the 2012 ap­proval for rheuma­toid arthri­tis and a 2017 OK for PsA.

Their no­tice to­day in­clud­ed this note:

The in­ter­im re­sults of the tri­al, as of Jan­u­ary 2019, have iden­ti­fied the fol­low­ing:

•19 cas­es of blood clots in the lung out of 3,884 pa­tient-years of fol­low-up in pa­tients who re­ceived to­fac­i­tinib 10 mg twice dai­ly, com­pared to 3 cas­es out of 3,982 pa­tient-years in pa­tients who re­ceived TNF block­ers

•45 cas­es of death from all caus­es out of 3,884 pa­tient-years of fol­low-up in pa­tients who re­ceived to­fac­i­tinib 10 mg twice dai­ly, com­pared to 25 cas­es out of 3,982 pa­tient-years in pa­tients who re­ceived TNF block­ers

The warn­ing on Xel­janz may well have a knock-on ef­fect at Eli Lil­ly, where agency con­cerns about Olu­mi­ant led them to steer clear of the high dose and stick with a low­er dose than the com­pa­ny felt would be ef­fec­tive. You can ex­pect the rest of the field to pay close at­ten­tion as they work on ri­val drugs — in­clud­ing some ther­a­pies they hope will have a bet­ter safe­ty pro­file.

So­cial im­age: Pfiz­er, AP Im­ages

CDC’s Robert Redford, NIAID’s Anthony Fauci, Admiral Brett Giroir at HHS, and FDA’s Stephen Hahn prepare to testify at a House hearing on June 23 (Getty)

'Ex­treme­ly po­lit­i­cal' — Trump neuters FDA's at­tempt to strength­en vac­cine EUA, ques­tions need to length­en process

Stephen Hahn went before a Senate committee Wednesday and declared he’s fighting. “Every one of the decisions we have reached has been made by career FDA scientists based on science and data, not politics,” he exclaimed, adding that “FDA will not permit any pressure from anyone to change that. I will fight for science.”

A few hours later, he was undermined by President Donald Trump when a reporter asked if he was okay with stricter vaccine guidelines that the FDA was said to be cooking up. “That has to be approved by the White House. We may or may not approve it. That sounds like a political move,” he decided.

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

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,500+ biopharma pros reading Endpoints daily — and it's free.

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.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,500+ biopharma pros reading Endpoints daily — and it's free.

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.

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.

Rep. Andy Harris (R-MD) (Tasos Katopodis/Pool via AP Images)

The mi­cro-cap that tapped a mask-skep­tic con­gress­man for their Covid DSMB is ap­ply­ing for an EUA. Their ev­i­dence? 21 pa­tients

NeuroRx, the tiny biotech that came under fire last week after Politico reported they selected a congressman and two other acquaintances of the CEO to supervise their Covid-19 drug trial, announced today that they will ask the FDA to authorize their drug based on the results of just 21 patients.

Such an application would test the agency’s standards of evidence for an EUA, which have already come under scrutiny after controversial authorizations for convalescent plasma and hydroxychloroquine. The only other company to discuss their intention to file for an EUA, Eli Lilly, did so after results came back from a randomized control study testing their antibody in over 450 patients.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,500+ biopharma pros reading Endpoints daily — and it's free.