FDA or­ders a par­tial hold on Epizyme’s lead can­cer drug tazeme­to­stat fol­low­ing T-cell lym­phoma case

Robert Baze­more, CEO Epizyme

Epizyme $EPZM is in some hot wa­ter this evening af­ter the biotech re­vealed a par­tial clin­i­cal hold on its cru­cial pro­gram for tazeme­to­stat.

The Cam­bridge, MA-based biotech was forced to slam the brakes on any en­roll­ment for its clin­i­cal tri­als of the drug for now as in­ves­ti­ga­tors sort out a pe­di­atric case of sec­ondary T-cell lym­phoma.

In­vestors didn’t like it. The biotech’s shares tanked 18% in af­ter-mar­ket trad­ing.

Says Epizyme:

Dos­es ex­plored in this study are high­er than those in the com­pa­ny’s Phase 2 adult stud­ies, an ap­proach not un­com­mon to drug de­vel­op­ment in ag­gres­sive, dif­fi­cult-to-treat pe­di­atric can­cers. At the time of the safe­ty re­port, the pa­tient had been on study for ap­prox­i­mate­ly 15 months and had achieved a con­firmed par­tial re­sponse. This pa­tient has now dis­con­tin­ued tazeme­to­stat and is be­ing treat­ed for T-cell lym­phoma. 

The EZH2 in­hibitor is be­ing stud­ied as a monother­a­py in on­go­ing Phase I and II pro­grams in mol­e­c­u­lar­ly de­fined sol­id tu­mors, in­clud­ing ep­ithe­lioid sar­co­ma and oth­er INI1-neg­a­tive tu­mors; both fol­lic­u­lar lym­phoma and dif­fuse large B-cell lym­phoma forms of NHL; mesothe­lioma; and com­bi­na­tion stud­ies in DL­B­CL and non-small cell lung can­cer.

Last sum­mer at AS­CO, as the biotech was look­ing to blaze an ac­cel­er­at­ed path­way to an ap­proval, in­ves­ti­ga­tors dis­ap­point­ed an­a­lysts with their lat­est set of re­spons­es in a Phase II for ep­ithe­lioid sar­co­ma.

“Pa­tient safe­ty is of the ut­most im­por­tance to Epizyme. We are work­ing ex­pe­di­tious­ly with clin­i­cal tri­al in­ves­ti­ga­tors and reg­u­la­to­ry au­thor­i­ties to ini­ti­ate the ap­pro­pri­ate steps to re­sume en­roll­ment,” said Robert Baze­more, pres­i­dent and chief ex­ec­u­tive of­fi­cer of Epizyme. “Epizyme, along with our glob­al in­ves­ti­ga­tor com­mu­ni­ty, has been very en­cour­aged by the clin­i­cal re­spons­es and tol­er­a­bil­i­ty of tazeme­to­stat ob­served in pe­di­atric and adult pa­tients with hema­to­log­i­cal ma­lig­nan­cies and sol­id tu­mors en­rolled in our tri­als. We re­main en­cour­aged by the po­ten­tial of tazeme­to­stat to ad­dress the un­met needs of many pa­tients liv­ing with can­cer.”

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.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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An­oth­er failed tri­al for Or­p­hazyme's 'pipeline-in-a-pro­duc­t' leaves shad­ow on drug's fu­ture

The tumultuous ride for Orphazyme continued on Friday as the company announced that a pivotal trial for its lead drug arimoclomol failed yet again, this time in the treatment of ALS, seeding doubt in a drug that had recently been cleared by the FDA for priority review. The latest failure casts a darker shadow on the upcoming decision despite Orphazyme’s upbeat outlook.

In a statement, the Danish biotech announced that the drug did not meet its primary or secondary endpoints evaluating function and survival. But the company has not announced any data surrounding the failure, instead saying that it will publish the complete results later this year.

Angela Merkel (AP Photo/Michael Sohn)

Covid-19 roundup: Pfiz­er sub­mits vac­cine for full ap­proval; Merkel op­pos­es Biden pro­pos­al to sus­pend IP for vac­cines

Pfizer and BioNTech said Friday that they’ve submitted a biologics license application to the FDA for full approval of their mRNA vaccine for those over the age of 16.

How long it will take the FDA to decide on the BLA will be set once it’s been formally accepted by the agency.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, previously told Endpoints News that the review of the BLA should take between three and four months, but it may be even faster than that.

As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

AstraZeneca’s closely-watched POSEIDON study continues to be the rare bright spot in its push for an in-house PD-L1/CTLA-4 combo.

Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

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In­cyte ponies up $12M to set­tle char­i­ty foun­da­tion kick­back claims; US ex­er­cis­es op­tion for more dos­es of mon­key­pox vac­cine

One in a string of lawsuits targeting copay charity foundations, the DOJ has been hunting drugmaker Incyte for what prosecutors alleged was a kickback scheme to court patients. Now, Incyte is clearing its name.

Incyte will shell out $12.6 million to settle claims it funneled funds through a charity foundation to cover federal copays for patients taking its JAK inhibitor Jakafi, the DOJ said this week.

Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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CEO Khurem Farooq (Gyroscope)

Hours be­fore ex­pect­ed de­but, Gy­ro­scope post­pones its IPO as 2 oth­er biotechs hold the line on their march to Nas­daq

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

In a surprising turn of events, UK-based Gyroscope Therapeutics has postponed its IPO mere hours before it was set to debut on Nasdaq.

Working on a gene therapy for wet AMD, Gyroscope was all set and ready to go public earlier this week, setting terms for a $142 million raise with a price range of $20 to $22. But in the wee hours of Friday morning, the company put out a press release saying they would delay their debut “in light of market conditions,” CEO Khurem Farooq said in a statement.

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Karyopharm taps long­time Pfiz­er, Am­gen vet to steer the ship; With Mer­ck in the rearview mir­ror, Roger Perl­mut­ter stakes his claim to a CEO job — and it's a sur­pris­ing choice

Like many who work in biopharma, Richard Paulson got started in the field because of a love of science.

Paulson had just finished business school and was looking to start a career that married his two passions. While looking for jobs, he thought of his grandmother who had struggled with Alzheimer’s disease, recalling how he saw first-hand what innovative medicines can do for patients. Ultimately, he started his first job in the space as a sales rep at Glaxo Wellcome, one of GlaxoSmithKline’s predecessors before its merger with SmithKline Beecham in 2000.