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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Alaa Halawaa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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'Band­ing to­geth­er': 50 fe­male biotech ex­ec­u­tives lay out plans for board di­ver­si­ty, new com­pa­nies and men­tor­ing founders

Earlier this month, during the Silicon Valley Bank meltdown, Angie You recalled the speed with which female biotech CEOs were helping each other connect with bankers, get their wires through and assuage concerns during a financial implosion.

This past weekend, 50 of about 125 women who are part of that Slack group and a broader coalition self-dubbed the Biotech Sisterhood met in person in Cancun for the second rendition of an annual summit connecting female biotech CEOs. The attendance list doubled that of the inaugural gathering in Arizona 12 months ago.

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FDA in­di­cates will­ing­ness to ap­prove Bio­gen ALS drug de­spite failed PhI­II study

Ahead of Wednesday’s advisory committee hearing to discuss Biogen’s ALS drug tofersen, the FDA appeared open to approving the drug, newly released briefing documents show.

Citing the need for flexibility in a devastating disease like ALS, regulators signaled a willingness to consider greenlighting tofersen based on its effect on a certain protein associated with ALS despite a failed pivotal trial. The documents come after regulatory flexibility was part of the same rationale the agency expressed when approving an ALS drug last September from Amylyx Pharmaceuticals, indicating the FDA’s openness to approving new treatments for the disease.

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No­var­tis touts sev­en years of dura­bil­i­ty da­ta for Zol­gens­ma

The same day that Roche touted positive durability and safety data for its spinal muscular atrophy drug Evrysdi, Novartis also made a splash with its multi-million dollar gene therapy for the disease.

Novartis rolled out interim data from two long-term follow-up studies Monday at the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference. In the first study, LT-001, all children in the trial that were treated after showing symptoms of SMA “maintained all previously achieved motor milestones” up to 7.5 years after being dosed. The average time since Zolgensma was given was 6.86 years.

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Rohan Palekar, 89bio CEO

89bio’s PhII da­ta add to quick suc­ces­sion of NASH read­outs as field seeks turn­around

89bio said its drug was better than placebo at lessening fibrosis without worsening nonalcoholic steatohepatitis, or NASH, in two of three dose groups.

The San Francisco biotech said it thinks the Phase IIb data pave the way for a potential Phase III, following in the footsteps of another biotech in its drug class, Akero Therapeutics. To fund a late-stage study, CEO Rohan Palekar told Endpoints News 89bio “would need to raise additional capital,” with the company having about $188 million at the end of last year.

Flare Therapeutics biochemists Yong Li (L) and Valerie Vivat

A $123M Flare will get Third Rock on­col­o­gy biotech in­to the clin­ic this year

Flare Therapeutics will start its first human trial this year with an investigational urothelial cancer drug after pulling together a $123 million Series B from Big Pharmas, VCs and its incubator, Third Rock Ventures.

Launched in 2021 on the idea that a biotech could finally succeed at drugging the much-sought-after but stubborn transcription factor, Flare Therapeutics said Wednesday it is now primed for the clinic after closing its large financing haul earlier this year. The raise is a relatively stark figure in a tough startup financing environment but further buoys the upbeat signals coming out of other Third Rock biotechs in recent weeks, including the $200 million CARGO Therapeutics and $100 million Rapport Therapeutics rounds.

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Francesco Marincola, newly-appointed Sonata Therapeutics CSO

Kite's head of re­search leaves for Flag­ship start­up Sonata

Another leader is departing Kite Pharma, and will to spend the “last part” of his career exploring how cancer evades the immune system.

Kite’s senior VP and global head of cell therapy research Francesco Marincola left the Gilead CAR-T unit last week for Sonata Therapeutics. Flagship last May unveiled the startup, which was pieced together from two fledgling biotechs Inzen and Cygnal Therapeutics. As CSO, Marincola will lead Sonata’s push to reprogram cancer cells to make them more immunogenic.

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NIH re­jects an­oth­er at­tempt to 'march-in' on Astel­las' prostate can­cer drug over ex­ces­sive price

The National Institutes of Health has again declined to use so-called “march-in” rights to lower the price of Astellas and Pfizer’s prostate cancer drug Xtandi despite being invented at UCLA with grants from the US Army and NIH.

“Given the remaining patent life and the lengthy administrative process involved for a march-in proceeding, NIH does not believe that use of the march-in authority would be an effective means of lowering the price of the drug,” NIH told prostate cancer patients Robert Sachs and Clare Love, in a letter shared with Endpoints News. The institutes’ analyses found Xtandi “to be widely available to the public,” an indication that there was not a pressing need for the US to act.