Once re­ject­ed, FDA now rolls out a short red car­pet for Am­i­cus’ mi­gala­s­tat

John Crow­ley

Am­i­cus CEO $FOLD John Crow­ley must have made a very fa­vor­able im­pres­sion on the FDA as he lob­bied se­nior of­fi­cials to over­turn their re­jec­tion of his ex­per­i­men­tal Fab­ry drug mi­gala­s­tat.

Not on­ly did reg­u­la­tors just ac­cept the fil­ing for a new re­view, the agency is giv­ing it pri­or­i­ty sta­tus, cut­ting the re­think to 6 months and flag­ging a clear in­di­ca­tion that it’s an odds-on fa­vorite for an of­fi­cial mar­ket­ing OK .

Cut­ting 4 months off the re­view process, the FDA will have its new de­ci­sion out by Au­gust 13.

An­a­lysts are al­ready count­ing the near-term rev­enue that Am­i­cus stands to earn. Joseph Schwartz at Leerink not­ed:

With the PDU­FA goal date of Aug 13, 2018, US rev­enue could con­tribute to FY2018 rev­enue guid­ance of $75M-$85M an­nounced last month. We con­tin­ue to see strong growth dri­vers for Am­i­cus this yr. as the com­pa­ny con­tin­ues to ad­vance its mi­gala­s­tat (Galafold; Fab­ry dis­ease) and ATB200/AT2221 (Pompe dis­ease) pro­grams through the clin­ic.

Crow­ley had em­ployed some du­bi­ous po­si­tions as he fought against the re­jec­tion, which came with a de­mand from the FDA for a new Phase III study of gas­troin­testi­nal symp­toms. Ini­tial­ly, the CEO had told in­vestors it would take 2 years to come up with the da­ta. But he told Janet Wood­cock and oth­ers that the ac­tu­al time­line would be 5 to 7 years, which made the de­vel­op­ment pro­gram im­prac­ti­cal.

Scott Got­tlieb

This was one of three drug pro­grams that the FDA re­versed it­self on in the months af­ter Scott Got­tlieb took over as com­mis­sion­er at the FDA, with Eli Lil­ly and Ther­a­peu­tic­sMD — chaired by ex-HHS sec­re­tary Tom­my Thomp­son — get­ting a do-over. The agency has nev­er ex­plained the re­jec­tions, nev­er ex­plained why it de­cid­ed to re­con­sid­er their de­ci­sion, or why it would of­fer Am­i­cus a quick de­ci­sion now.

But they nev­er do, re­quired by the rules to stay qui­et. Got­tlieb had ini­tial­ly said he would press for the redact­ed pub­li­ca­tion of com­plete re­sponse let­ters like these, but he re­cent­ly shrugged that off.

“Con­grat­u­la­tions, again!” Crow­ley wrote in a note to Got­tlieb at­tached to his case for a re­view. “As dis­cussed, the hopes and well be­ing of so many liv­ing with rare, dev­as­tat­ing dis­eases rests now with your great lead­er­ship and wis­dom. Thanks for your at­ten­tion to the at­tached.”

Got­tlieb’s staff told me that the com­mis­sion­er nev­er saw that pack­et, redi­rect­ing it to oth­er se­nior of­fi­cials at the FDA. Of­fi­cials, though, de­clined to ex­plain what Crow­ley meant by the word “again,” or if Got­tlieb had dis­cussed the is­sue with the CEO.

That note, along with Crow­ley’s as­ser­tions on the time­line to re­spond, were ob­tained un­der FOIA by the Pro­ject on Gov­ern­ment Over­sight.

Got­tlieb has open­ly ad­vo­cat­ed for a more ef­fi­cient re­view process, par­tic­u­lar­ly where rare dis­ease are con­cerned. That al­so squares with Pres­i­dent Don­ald Trump’s vow to speed up the reg­u­la­to­ry process. And af­ter start­ing out with a high rat­ing from the in­dus­try, biotech ex­ecs are now cheer­ing him on as one of the best things that’s hap­pened at the FDA in years.

Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

When will it end? Gold­man econ­o­mist gives late-stage vac­cines a good shot at tar­get­ing 'large shares' of the US by mid-2021 — but the down­side is daunt­ing

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can create herd immunity in the US by the middle of next year, with Europe following a few months later.

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Sean Nolan and RA Session II

Less than 3 months af­ter launch, the AveX­is crew’s Taysha raise $95M Se­ries B. Is an IPO next?

The old AveXis team is moving quickly in Dallas.

Three months ago, they launched Taysha with $30 million in Series A funding and a pipeline of gene therapies out of UT Southwestern. Now, they’ve announced an oversubscribed $95 million Series B. And the biotech is declining all interview requests on the news, the kind of broad silence that can indicate an IPO is in the pipeline.

Biotechs, including those relatively fresh off launch, have been going public at a frenzy since the pandemic began. Investors have showed a willingness to put upwards of $200 million to companies that have yet to bring a drug into the clinic. Still, if Taysha were to go public in the near future, it would be perhaps the shortest path from launch to IPO in recent biotech memory.

UP­DAT­ED: No­vavax her­alds the lat­est pos­i­tive snap­shot of ear­ly-stage Covid-19 vac­cine — so why did its stock briefly crater?

High-flying Novavax $NVAX became the latest of the Covid-19 vaccine players to stake out a positive set of biomarker data from its early-stage look at its vaccine in humans.

Their adjuvanted Covid-19 vaccine was “well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera,” the company noted. According to the biotech:

All subjects developed anti-spike IgG antibodies after a single dose of vaccine, many of them also developing wild-type virus neutralizing antibody responses, and after Dose 2, 100% of participants developed wild-type virus neutralizing antibody responses. Both anti-spike IgG and viral neutralization responses compared favorably to responses from patients with clinically significant COVID‑19 disease. Importantly, the IgG antibody response was highly correlated with neutralization titers, demonstrating that a significant proportion of antibodies were functional.

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Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

Mod­er­na CEO Stéphane Ban­cel out­lines a prospec­tive moth­er­lode of Covid-19 vac­cine rev­enue — will a back­lash fol­low?

Moderna shows no sign of slowing down, or turning charitable when it comes to pricing supplies of its Covid-19 vaccine.

One of the leaders in the Phase III race to get a Covid-19 vaccine across the finish line in record time, Moderna says it’s on track to complete enrollment in one of the most avidly watched studies in the world next month. And the biotech has already banked some $400 million in deposits for vaccine supply as it works through negotiations with countries around the world — as CEO Stéphane Bancel sets out to hire a commercial team.

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Paul Laikind, ViaCyte CEO

Stem cell play­er Vi­a­Cyte ex­pands col­lab­o­ra­tion with Gore to de­vel­op sub­cu­ta­neous di­a­betes treat­ment

Longtime stem cell player ViaCyte has teamed up with a materials science company in an effort to solve immunosuppression challenges and advance its type 1 diabetes treatments.

Expanding on an existing collaboration, ViaCyte and W.L. Gore have agreed to combine the biotech’s PEC-Encap candidate with a Gore-produced membrane in what they hope will eliminate the need for immunosuppressive drugs. Such treatments have created foreign body responses in the past, and stamping these reactions out is the main goal, ViaCyte CEO Paul Laikind said.

My­ovant lands a fresh $200M loan as FDA mar­ket­ing de­ci­sion looms; Amarin goes it alone in Eu­rope

Myovant is getting ready to roll out its commercial operations to back relugolix, now under FDA review for prostate cancer.

The startup has added a fresh $200 million in support from Sumitomo Dainippon Pharma, which controls a majority of the stock $MYOV. Sumitomo is handing the cash over as a loan, bringing its total to $600 million. Myovant — which is gearing up for a showdown with AbbVie — has also filed an NDA to sell relugolix for uterine fibroids and recently posted positive late-stage data for endometriosis.

J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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Covid-19 roundup: J&J and BAR­DA agree to $1 bil­lion for 100 mil­lion dos­es; Plas­ma re­duces mor­tal­i­ty by 50% — re­ports

J&J has become the latest vaccine developer to agree to supply BARDA with doses of their Covid-19 vaccine, signing an agreement that will give the government 100 million doses in exchange for $1 billion in funding.

The agreement, similar to those signed by Novavax, Sanofi and AstraZeneca-Oxford, provides funding not only for individual doses but to help J&J ramp up manufacturing. Pfizer, by contrast, received $1.95 billion for the doses alone. Still, if one looked at each agreement as purchase amounts, J&J’s deal would be $10 per dose, slotting in between Novavax’s $16 per dose and AstraZeneca’s $4 per dose.

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Ver­sant de­buts Ridge­line's start­up #4, armed with $30M and al­ter­na­tive TCR cell ther­a­pies for sol­id tu­mors

For all the iterations and advances in TCR therapies for cancer, any experimental treatments involving T cell receptors share one trait: By definition, they only recognize antigens presented as peptides on the major histocompatibility complex (MHC) on cells.

Versant reckons it’s time to expand the arsenal. With $30 million in initial funding, its Ridgeline Discovery Engine in Switzerland has been working on a non-peptidic approach that it says has tumor-agnostic potential, especially in solid tumors. They’ve named it Matterhorn, after a Swiss mountain as they did with the three other companies that have emerged from the Basel-based incubator.