XBiotech shares crater as CHMP shud­ders at its ap­pli­ca­tion to sell Xilonix

It’s pos­si­ble Austin-based XBiotech just doesn’t get it.

The EMA’s Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use — the CHMP — put to­geth­er an in­for­mal trend vote on the biotech’s ap­pli­ca­tion for Xilonix as a new drug for col­orec­tal can­cer, pro­vid­ing a hard sig­nal the for­mal vote will go against XBiotech af­ter con­clud­ing the biotech has no da­ta to prove that the drug works.

Like­ly out­come: out­right re­jec­tion. XBiotech’s shares cratered, drop­ping 35% on its lat­est set­back, which has been com­ing hard and fast.

Back in late 2015 XBiotech out­lined a se­ries of em­bar­rass­ing prat­falls that oc­curred in their first Phase III tri­al, with a batch of pa­tients in the drug and place­bo arm get­ting the wrong dose, 25 drop­ping out be­fore the study be­gan and 33 more un­evalu­able be­cause they weren’t prop­er­ly eval­u­at­ed for a re­sponse — an er­ror rate of 25% in the pop­u­la­tion.

The Eu­ro­pean reg­u­la­tors con­clud­ed that the clin­i­cal rel­e­vance was un­der­whelm­ing with “no sup­port­ive ev­i­dence of ef­fi­ca­cy.”

Not what you want to hear.

Nev­er­ther­less, XBiotech sig­naled it might ap­peal in CEO John Simard’s pre­pared re­sponse: “We are dis­ap­point­ed by the out­come of the meet­ing. We be­lieve that the da­ta speak in a clear and re­sound­ing voice to clin­i­cal rel­e­vance of a new an­ti­body ther­a­py in ad­vanced col­orec­tal can­cer. We be­lieve that find­ings from our Phase III study show that we have de­vel­oped an im­por­tant end­point and method­ol­o­gy to eval­u­ate an­ti-can­cer ther­a­py in ad­vanced stage dis­ease and that our mon­o­clon­al an­ti­body rep­re­sents a break­through treat­ment in pa­tients with ad­vanced col­orec­tal can­cer. The EMA mar­ket­ing au­tho­riza­tion ap­pli­ca­tion pro­ce­dure en­ables the ap­peal of neg­a­tive de­ci­sions from the oral ex­pla­na­tion. We may seek ac­cess to this process at the ap­pro­pri­ate time.”

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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FDA ex­tends re­search agree­ment with MIT-li­censed or­gan-on-chip sys­tems

The FDA on Wednesday extended its four-year agreement with CN Bio, a developer of single- and multi-organ-on-chip systems used for drug discovery, for another three years.

CN Bio said the scope of the research performed by the FDA’s Center for Drug Evaluation and Research has expanded to include the exploration of the company’s lung-on-a-chip system to help with the agency’s evaluation of inhaled drugs, in addition to the agency’s work on its liver model.

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In quest to meet user fee goals, FDA’s per­for­mance con­tin­ues down­ward trend

A recent update to the FDA’s running tally of how it’s meeting its user fee-related performance goals during the pandemic shows an agency that is not out of the woods yet.

The latest numbers reveal that for a second straight quarter in 2021, the FDA has met its user fee goal dates for 93% of original new drug applications, which compares with 94% and 98% for the previous two quarters in 2020, respectively.

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UP­DAT­ED: Pfiz­er hits the brakes on their piv­otal tri­al for a BC­MA/CD3 bis­pe­cif­ic on safe­ty con­cerns while FDA road­block is hold­ing up Duchenne MD PhI­II

Pfizer’s ambitious plan to take a Phase II study of its BCMA CD3-targeted bispecific antibody elranatamab (PF-06863135) and run it through to an accelerated approval has derailed.

The pharma giant said in a release this morning that they have halted enrollment for their MagnetisMM-3 study after researchers tracked three cases of peripheral neuropathy in the ongoing Phase I. They are now sharing info with the FDA as they explore the red safety flag.

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Thomas Schall, ChemoCentryx CEO (file photo)

Chemo­Cen­tryx plunges as FDA rais­es ques­tions about rare dis­ease drug ahead of ad­comm

ChemoCentryx’s stock price on Wednesday was cut in half by the release of FDA briefing documents ahead of a Thursday adcomm, raising questions on the company’s clinical data to support avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

ANCA-associated vasculitides (AAV) affect small to medium-size blood vessels that can be fatal in less than a year if left untreated, according to FDA. Only Roche’s Rituxan is currently FDA-approved for the treatment of AAV, while glucocorticoids are approved for the broader indication of vasculitis.

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Re­gen­eron’s gold­en goose Eylea may stave off biosim­i­lar com­pe­ti­tion un­til 2024 or be­yond

Almost 10 years have passed since its first FDA approval and Regeneron’s macular degeneration injection Eylea continues to pile up sales to the tune of about $5 billion per year, or more than half of Regeneron’s annual revenues.

Those billions are not expected to go anywhere anytime soon thanks to competition, even as Novartis subsidiary Sandoz announced Monday that it’s beginning a Phase III trial for an Eylea biosimilar in 460 patients across 20 countries.

Cynthia Butitta (L) and Joe Jimenez

Is that an­oth­er IPO in the mak­ing? Ex-No­var­tis CEO Joe Jimenez and a lead Kite play­er take up new posts at an off-the-shelf ri­val to 2 pi­o­neer­ing drugs

Right on the heels of taking on a $160 million crossover round in a likely leap to Nasdaq, Century Therapeutics CEO Lalo Flores is now pushing ahead with the high-profile ex-Novartis chief Joe Jimenez as chairman.

Jimenez’s greatest fame at Novartis was earned for one of its weakest products, as their pioneering personalized CAR-T Kymriah won the honors for the first such drug to make it to the market. Now a host of players, including Century, are barreling in behind the frontrunners with allogeneic rivals that can be created for off-the-shelf use.

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An em­ploy­ee com­plaint at Eli Lil­ly's Branch­burg plant al­leges al­tered qual­i­ty con­trol docs amid FDA probe — re­port

Eli Lilly was one of the earliest players in the race for a Covid-19 antibody, but a series of setbacks at a New Jersey manufacturing site have set back its efforts. Now, an internal complaint reportedly claims that a director at that site knowingly fudged quality control docs right under the FDA’s nose.

An employee complaint from Eli Lilly’s manufacturing plant in Branchburg, NJ, alleged that a director altered documents handed over to FDA regulators as part of an effort to downplay serious quality control issues amid the agency’s probe at the site, Reuters reported.