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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

UP­DAT­ED: Boehringer nabs FDA's first in­ter­change­abil­i­ty des­ig­na­tion for its Hu­mi­ra com­peti­tor — but will it mat­ter?

The FDA late Friday awarded Boehringer Ingelheim the first interchangeability designation for its Humira biosimilar Cyltezo, meaning that when it launches in July 2023, pharmacists will be able to automatically substitute the Boehringer’s version for AbbVie’s mega-blockbuster without a doctor’s input.

The designation will likely give Boehringer, which first won approval for Cyltezo in 2017, the leg up on a crowded field of Humira competitors.

Bio­gen hit by ALS set­back with PhI­II fail­ure for tofersen — but fol­lows a fa­mil­iar strat­e­gy high­light­ing the pos­i­tive

Patients and analysts waiting to hear Sunday how Biogen’s SOD1-ALS drug tofersen fared in Phase III didn’t have to wait long for the top-line result they were all waiting for. The drug failed the primary endpoint on significantly improving the functional and neurologic decline of patients over 28 weeks as well as the extension period for continued observation.

In fact, there was very little difference in response.

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Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Jeffrey Nau, Oyster Point Pharma CEO

FDA OKs an in­haled ver­sion of smok­ing ces­sa­tion drug Chan­tix — for a com­mon eye dis­ease

Oyster Point Pharma now has its first FDA-approved product — Tyrvaya. And the biotech has taken a unique route to get there by using an old drug with a storied past.

The New Jersey biotech announced this morning that the FDA has approved their nasal spray product for dry eye disease on Friday — the first nasal spray to be approved for the disease. The product’s active ingredient is 0.03 mg of varenicline, also known as smoking cessation aid Chantix.

Omeros plunges deep­er af­ter FDA re­jects rare dis­ease drug, ask­ing for more in­for­ma­tion

Omeros practically warned investors that a complete response letter was coming when it disclosed that the FDA found deficiencies in its BLA for narsoplimab. But the agency did not elaborate on the specifics of those deficiencies for the drug, which was being positioned as a treatment for the rare but serious blood clotting disease known as hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA).

UP­DAT­ED: Third time's the charm: Adamis nabs ap­proval of high-dose nalox­one in­jec­tion af­ter two CRLs

If at first you don’t succeed, and at second you don’t succeed, try, try again.

That’s been the unofficial mantra for Adamis Pharmaceuticals’ high dose naloxone injection, which received two CRLs in the span of a year between the Novembers of 2019 and 2020. But on Monday, word came through that the FDA approved the drug on Adamis’ third attempt, giving doctors another tool to treat individuals who overdose on opioids.

Two drug­mak­ers hit with PDU­FA date de­lays from FDA amid back­log of in­spec­tions

As the FDA is weighed down with more and more pandemic responsibilities, the agency is beginning to miss PDUFA dates with more frequency too. Two different companies on Monday said they received notices that the FDA has not completed their drug reviews on time.

The review of an NDA for Avadel Pharmaceuticals’ candidate treatment for narcolepsy is not coming this month, the company said, and the review of UCB’s BLA for bimekizumab, used to treat moderate to severe plaque psoriasis, will miss its target date as well.

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Reshma Kewalramani, Vertex CEO (YouTube)

Ver­tex gets much-need­ed win with ‘ex­tra­or­di­nary’ first pa­tient re­sults on po­ten­tial di­a­betes cure

Vertex said Monday that the first patient dosed with its cell therapy for type 1 diabetes saw their need for insulin injections vanish almost entirely, a key early step in the decades-long effort to develop a curative treatment for the chronic disease.

The patient, who had suffered five potentially life-threatening hypoglycemic — or low blood sugar — episodes in the year before the therapy, was injected with synthetic insulin-producing cells. After 90 days, the patient’s new cells produced insulin steadily and ramped up their insulin production after a meal like normal cells do, as measured by a standard biomarker for insulin production.

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