Epizyme hits the ground run­ning with sec­ond — and cru­cial — OK for Tazverik, in fol­lic­u­lar lym­phoma

When Epizyme scored the first-ever ap­proval for tazeme­to­stat, in ep­ithe­lioid sar­co­ma, ex­ecs made it clear it was on­ly an ap­pe­tiz­er, some­thing to whet the ap­petites of reg­u­la­tors, in­vestors and physi­cians to the po­ten­tial of the methyl­trans­ferase in­hibitor. Five months lat­er, they are ready to serve the main course.

She­fali Agar­w­al

The biotech got all the help from the FDA to ex­pand the la­bel for Tazverik, go­ing through pri­or­i­ty re­view to land an ac­cel­er­at­ed ap­proval in two in­di­ca­tions of fol­lic­u­lar lym­phoma:

  • Adult pa­tients with re­lapsed or re­frac­to­ry FL whose tu­mors are pos­i­tive for an EZH2 mu­ta­tion as de­tect­ed by an FDA-ap­proved test and who have re­ceived at least two pri­or sys­temic ther­a­pies
  • Adult pa­tients with re­lapsed or re­frac­to­ry FL who have no sat­is­fac­to­ry al­ter­na­tive treat­ment op­tions

The OK was an­chored on over­all re­sponse rate and du­ra­tion of re­sponse in a Phase II, which en­com­passed both mu­tat­ed and wild-type EZH2.

“As a re­minder, in the EZH2 MT co­hort, the re­sponse rate was 69%, while in the EZH2 WT co­hort, the re­sponse rate was rough­ly half, at 35%,” SVB Leerink an­a­lyst An­drew Berens wrote at the news of the pri­or­i­ty re­view. “While we think that the ben­e­fit in the wild-type pa­tients is still com­pelling enough for some pa­tients to con­sid­er ther­a­py, it is less com­pelling than that seen in the EZH2m co­hort.”

Robert Baze­more

That could mean that in prac­tice, clin­i­cians will want a EZH2 test be­fore pre­scrib­ing the drug, re­duc­ing the sig­nif­i­cance of the broad la­bel.

As such, he mod­els $17.5 mil­lion in FL sales and $5.6 mil­lion of ES sales for 2020.

CMO She­fali Agar­w­al, though, is clear­ly pleased with what they achieved with the “very ef­fi­cient” NDA, lever­ag­ing da­ta that have al­ready been sub­mit­ted with the ES pack­age. Es­pe­cial­ly con­sid­er­ing that FL, a sub­type of non-Hodgkin lym­phoma that ac­counts for 20% to 30% of cas­es, is of­ten con­sid­ered a chron­ic dis­ease.

“In our view, there re­mains no clear stan­dard of care in the re­lapsed and/or re­frac­to­ry FL pop­u­la­tion as not all pa­tients ben­e­fit from to­day’s avail­able ther­a­pies,” she said in a state­ment. “Based on this la­bel, physi­cians will have the abil­i­ty to use their clin­i­cal dis­cre­tion to pre­scribe TAZVERIK for their re­lapsed or re­frac­to­ry pa­tients re­gard­less of EZH2 mu­ta­tion­al sta­tus and with­out re­gard to a spe­cif­ic line of treat­ment where oth­er op­tions are not sat­is­fac­to­ry.”

CEO Robert Baze­more added that Epizyme has al­ready “seam­less­ly” ex­pand­ed the com­mer­cial team, armed with what they be­lieve is a no­table safe­ty pro­file and com­pelling op­tion for oral, at-home ad­min­is­tra­tion.

A full ap­proval will hinge up­on a 500-pa­tient con­fir­ma­to­ry tri­al Epizyme is con­duct­ing that pairs Tazverik with Revlim­id and rit­ux­imab (Rit­ux­an) for FL pa­tients in the sec­ond or lat­er lines.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Ex­elix­is pulls a sur­prise win in thy­roid can­cer just days ahead of fi­nal Cabome­tyx read­out

Exelixis added a thyroid cancer indication to its super-seller Cabometyx’s label on Friday — months before the FDA was expected to make a decision, and days before the company was set to unveil the final data at #ESMO21.

At a median follow-up of 10.1 months, differentiated thyroid cancer patients treated with Cabometyx (cabozantinib) lived a median of 11 months without their disease worsening, compared to just 1.9 months for patients given a placebo, Exelixis said on Monday.

Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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Ex-My­lan em­ploy­ee pleads guilty to in­sid­er trad­ing, il­le­gal­ly deal­ing on FDA ap­provals, earn­ings and Up­john merg­er

A former Mylan IT executive pleaded guilty Friday to an insider trading scheme where he bought and sold stock options on another executive’s advice.

Prosecutors secured the plea from Dayakar Mallu, Mylan’s former VP of global operations information technology, after uncovering the plan. Mallu collaborated with an unnamed “senior manager,” the SEC said, to trade options ahead of Mylan public announcements regarding FDA approvals, revenue reports and its merger with the Pfizer generics subsidiary Upjohn. The two subsequently shared profits.

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Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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Rafaèle Tordjman (Jeito Capital)

Con­ti­nu­ity and di­ver­si­ty: Rafaèle Tord­j­man's women-led VC firm tops out first fund at $630M

For a first-time fund, Jeito Capital talks a lot about continuity.

Rafaèle Tordjman had spotlighted that concept ever since she started building the firm in 2018, promising to go the extra mile(s) with biotech entrepreneurs while pushing them to reach patients faster.

Coincidentally, the lack of continuity was one of the sore spots listed in a report about the European healthcare sector published that same year by the European Investment Bank — whose fund is one of the LPs, alongside the American pension fund Teacher Retirement System of Texas and Singapore’s Temasek, to help Jeito close its first fund at $630 million (€534 million). As previously reported, Sanofi had chimed in €50 million, marking its first investment in a French life sciences fund.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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