Af­ter fac­ing a 16-month so­journ dur­ing Covid to over­come a stun­ning CRL, lit­tle En­zy­vant says it's back on track — ex­pects mar­ket­ing de­ci­sion lat­er this year

Back in late 2019, En­zy­vant CEO Rachelle Jacques was shocked when the FDA is­sued a CRL for its ther­a­py to treat ul­tra-rare cas­es of pe­di­atric con­gen­i­tal athymia — a con­di­tion marked by ex­treme im­mune de­fi­cien­cy and an ear­ly death. Like a lot of old­er, big­ger or­ga­ni­za­tions, Jacques dis­cov­ered that reg­u­la­tors were not ea­ger to OK the CMC set­up at En­zy­vant, though she told me at the time that she be­lieved that with no de­mands for new clin­i­cal da­ta, she felt that it could all be re­solved in “a few months.”

That was 16 months ago.

To­day, Jacques is putting out word that En­zy­vant has an­swered all the FDA’s chal­lenges and put their re­sub­mit­ted BLA for RVT-802 back on track for a PDU­FA dead­line of Oc­to­ber 8 — close to 2 years af­ter the stun­ning set­back.

So what hap­pened?

First, she and her team de­cid­ed to pre­pare care­ful­ly to set up their Type A meet­ing in March of last year to get a de­tailed read on ex­pec­ta­tions.

“Re­al­ly, when com­ing out of that meet­ing then the work be­gan,” says the CEO. “The meet­ing was meant to be face-to-face but it was about a week or so af­ter the shut­down start­ed. We were lucky that the agency piv­ot­ed that to a vir­tu­al meet­ing. It hit all the marks and we got what we need­ed out of it.”

So, what, ex­act­ly, did they have to do to re­solve the CRL?

“We did need to do some fa­cil­i­ties con­struc­tion and we did that dur­ing Covid,” says Jacques. “That’s some­thing that we didn’t an­tic­i­pate im­me­di­ate­ly when we had the let­ter in our hands, but cer­tain­ly as we talked with the agency that was very clear that was a so­lu­tion. A lot of the oth­er work was re­al­ly just doc­u­ment­ing at a very gran­u­lar lev­el some of the process­es and so on as well as some ad­di­tion­al stud­ies we did to sup­ple­ment the da­ta we pro­vid­ed on the over­all man­u­fac­tur­ing process — and those were long lead time items as well.”

En­zy­vant was a cre­ation of Roivant in­volved in the big Sum­i­to­vant buy­out and ini­tial­ly was in the lead po­si­tion for de­liv­er­ing the first ap­proval for Vivek Ra­maswamy’s group. That first timer hon­or went to My­ovant as En­zy­vant was forced back in the line. Jacques isn’t say­ing how big the team is at En­zy­vant af­ter work­ing through the re­sub­mis­sion, on­ly go­ing with “un­der 50” to in­di­cate size.

Safe to say, it’s tiny.

The biotech has side­lined its one oth­er drug pro­gram, an en­zyme re­place­ment ther­a­py, shop­ping it to any bid­der in­ter­est­ed in tak­ing it on. And the CEO says the team will stay 100% fo­cused on a ther­a­py that cov­ers some 20 in­fants a year. Pric­ing hasn’t been es­tab­lished yet, she adds, but not­ing the com­plex­i­ty and trau­ma as­so­ci­at­ed with treat­ing these in­fants with no cur­rent­ly ap­proved drug, it doesn’t sound like it will break ranks with a com­mon in­dus­try for­mu­la to go high­er as pop­u­la­tions shrink.

Next steps in­clude a look be­yond Amer­i­ca’s bor­ders.

Mov­ing Out of the Clin­ic with Dig­i­tal Tools: Mo­bile Spirom­e­try Dur­ing COVID-19 & Be­yond

An important technology in assessing lung function, spirometry offers crucial data for the diagnosis and monitoring of pulmonary system diseases, as well as the ongoing measurement of treatment efficacy. But trends in the healthcare industry and new challenges introduced by the COVID-19 pandemic are causing professionals in clinical practice and research to reevaluate spirometry’s deployment methods and best practices.

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Sanofi, Glax­o­SmithK­line jump back in­to the PhI­II race for a Covid vac­cine — as the win­ners con­gre­gate be­hind the fin­ish line

Sanofi got out early in the race to develop a vaccine using more of a traditional approach, then derailed late last year as their candidate failed to work in older people. Now, after likely missing the bus for the bulk of the world’s affluent nations, they’re back from that embarrassing collapse with a second attempt using GSK’s adjuvant that may get them back on track — with a potential Q4 launch that the rest of the world will be paying close attention to.

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SCO­TUS de­clines to re­view En­brel biosim­i­lar case, tee­ing up 30+ years of ex­clu­siv­i­ty and $20B more for Am­gen’s block­buster

As the House Oversight Committee is set to grill AbbVie CEO Richard Gonzalez on Tuesday over tactics to block competition for its best-selling drug of all time, another decision on Capitol Hill on Monday opened the door for billions more in Amgen profits over the next eight years.

The Supreme Court on Monday denied Novartis subsidiary Sandoz’s petition to review a Federal Circuit’s July 2020 decision concerning its biosimilar Erelzi (etanercept-szzs), which FDA approved in 2016 as a biosimilar to Amgen’s Enbrel (etanercept). Samsung’s Enbrel biosimilar Eticovo also won approval in 2019 and remains sidelined.

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How to man­u­fac­ture Covid-19 vac­cines with­out the help of J&J, Pfiz­er or Mod­er­na? Bi­ol­yse sees the dif­fi­cul­ties up close

When Biolyse, an Ontario-based manufacturer of sterile injectables, forged a deal with Bolivia last week to manufacture up to 50 million J&J Covid-19 vaccine doses, the agreement kicked off what will prove to be a test case for how difficult the system of compulsory licenses is to navigate.

The first problem: When Biolyse asked J&J, via a March letter, to license its Covid-19 vaccine, manufacture it in Canada and pay 5% royalties on shipments to needy, low-income countries, J&J rejected the offer, refusing to negotiate. J&J also did not respond to a request for comment.

Tim Mayleben (L) and Sheldon Koenig (Esperion)

On the heels of a sting­ing Q1 set­back, Es­pe­ri­on's long­time cham­pi­on is ex­it­ing the helm and turn­ing the wheel over to a mar­ket­ing pro

Just days after getting stung by criticism from a badly disappointed group of analysts, there’s a big change happening today at the helm of Esperion $ESPR.

Longtime CEO Tim Mayleben, who championed the company for 9 years from early clinical through a lengthy late-stage drive to successfully get their cholesterol drug approved for a significant niche of patients in the US, is out of the C suite, effective immediately. Sheldon Koenig — hired at the end of 2020 with a resume replete with Big Pharma CV sales experience —  is stepping into his place, promising to right a badly listing commercial ship that’s been battered by market forces.

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No­var­tis' En­tresto takes its 2nd fail­ure of the week­end at ACC, show­ing no ben­e­fit in most dire heart fail­ure pa­tients

Novartis’ Entresto started the ACC weekend off rough with a trial flop in heart attack patients, slowing the drug’s push into earlier patients. Now, an NIH-sponsored study is casting doubt on Entresto’s use in the most severe heart failure patients, another black mark on the increasingly controversial drug’s record.

Entresto, a combination of sacubitril and valsartan, could not beat out valsartan alone in an outcomes head-to-head for severe heart failure patients with a reduced ejection fraction (HFrEF), according to data presented Monday at the virtual American College of Cardiology meeting.

A T-cell play­er with back­ing from Roche takes next big step for BiTE drugs with 'on-of­f' switch to avoid tox­i­c­i­ty

The bispecific T cell engager field is absolutely packed with big-name players who have crowded in despite some high-profile failures in the class. Now, a Bay Area biotech thinks it may have the key to tackling BiTE toxicity, using an old “on-off switch” idea to give doctors more control of the drugs’ effect on patients.

San Francisco-based Soteria Biotherapeutics uncloaked Monday with a $42 million Series A co-led by Roche Venture Fund and 5AM Ventures with participation from the Novartis Venture Fund to advance its bispecific T cell engagers with an “on-off” switch the founders think can avoid some of the dire safety flags endemic to the class.

Matt Gline (L) and Vivek Ramaswamy

In­sid­er ac­count of Roivan­t's SPAC deal — and that $7.3B val­u­a­tion — re­veals a few se­crets as Matt Gline po­si­tions the com­pa­ny as the new ‘Big Phar­ma’

It was Oct. 7, 2020, and Matt Gline wasn’t wasting any time.

The CEO of Roivant had word that KKR vet Jim Momtazee’s SPAC had priced late the night before, triggering a green light for anyone interested in pursuing a big check for future operations and riding the financial instrument to Nasdaq. So he wrote a quick email congratulating Momtazee, whom he knew, for the launch.

Oh, and maybe Momtazee would like to schedule something with Gline and his executive chairman, Roivant founder Vivek Ramaswamy, to chat about Roivant and its business?

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$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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