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.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

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De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Papa (Ryan Remiorz/The Canadian Press via AP, File)

Joe Pa­pa re­signs as chair of Bausch Health as bil­lion­aire John Paul­son takes over

Joe Papa, chair of Bausch Health, officially resigned on Thursday and the board appointed billionaire hedge fund manager John Paulson as the new chair, effective immediately.

The specialty pharma company sought to make clear that Papa’s abrupt departure “was not due to any dispute or disagreement with the Company, its management or the Board on any matter relating to the Company’s operations, policies or practices.”

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Alarmed by side ef­fect, FDA slaps clin­i­cal hold on Sarep­ta's next-gen Duchenne drug

Sarepta Therapeutics’ next-gen Duchenne muscular dystrophy drug has been hit with a clinical hold after investigators flagged a serious case of low magnesium levels in one patient’s blood.

Screening and dosing will be halted in what is known as Part B of the Phase II MOMENTUM study, which has enrolled about half of the planned patients. Sarepta said it will be submitting information on all cases of the condition, known as hypomagnesemia, per the FDA’s request and proposing some changes to the risk mitigation and safety monitoring plan.

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