John Crowley, Amicus chairman and CEO

In an­tic­i­pa­tion of PhI­II tri­al re­sults, Am­i­cus pares down old debt with $400 mil­lion loan

Am­i­cus re­ceived a healthy heap­ing of good news last month when an­a­lysts threw cold wa­ter on Sanofi’s late-on­set Pompe dis­ease ERT, af­ter a Phase III study es­sen­tial­ly cleared the way for Am­i­cus’ com­pet­ing can­di­date to be the stan­dard-bear­er. Now, Am­i­cus is prepar­ing for a new “path to prof­itabil­i­ty” with an ex­pect­ed wind­fall.

The New Jer­sey-based bio­phar­ma an­nounced to­day it is re­fi­nanc­ing debt to the tune of a $400 mil­lion loan from Hayfin Cap­i­tal Man­age­ment. Am­i­cus will uti­lize the cred­it, which is ex­pect­ed to be fund­ed in full on or about Au­gust 4, to re­fi­nance ex­ist­ing debt and for oth­er de­vel­op­ment pur­pos­es.

As part of the an­nounce­ment, Am­i­cus al­so not­ed it re­mains on track to see be­tween $250 and $260 mil­lion in rev­enue from sales of its drug Galafold, which treats Fab­ry dis­ease.

Cru­cial­ly for Am­i­cus, the loan al­lows cur­rent oblig­a­tions to be paid with­out turn­ing to the eq­ui­ty mar­ket and di­lut­ing its own­er­ship stake. In oth­er words, Am­i­cus did not have to sell off shares to sub­si­dize debt pay­ments.

Am­i­cus will on­ly need to pay in­ter­est un­til mid-2024. Then, be­gin­ning on the four-year an­niver­sary of the loan with­draw­al, the com­pa­ny will re­pay the prin­ci­pal amount in nine equal quar­ter­ly in­stall­ments un­til the loan ma­tures in late 2026, ac­cord­ing to an SEC fil­ing. Am­i­cus can al­so pay back the en­tire prin­ci­pal amount at any time.

The mere fact that Hayfin is will­ing to pro­vide such a large line of cred­it il­lus­trates the firm’s con­fi­dence in Am­i­cus’ abil­i­ty to not on­ly re­pay in full, but in the up­com­ing Pompe dis­ease can­di­date it­self. Since the re­lease of Sanofi’s study last month, Am­i­cus stock has risen about 40 per­cent.

That study showed un­der­whelm­ing da­ta for Sanofi’s neo-GAA pro­gram in treat­ing Pompe dis­ease when com­pared to Lu­mizyme, an ear­li­er-gen­er­a­tion ERT from Sanofi. Though neo-GAA met its pri­ma­ry end­point in non-in­fe­ri­or­i­ty, it did not demon­strate su­pe­ri­or­i­ty, a Cowen an­a­lyst said at the time.

Ad­di­tion­al­ly, a cross-tri­al com­par­i­son from SVB Leerink in­di­cat­ed that Sanofi’s Phase III re­sults showed less ef­fi­ca­cy than Am­i­cus’ Phase II re­sults. Am­i­cus is cur­rent­ly con­duct­ing its Phase III tri­al for its Pompe dis­ease can­di­date, AT-GAA, and plans to ap­ply for and com­plete a rolling BLA some­time in the first half of next year.

The com­ple­tion of the Am­i­cus Phase III could pro­vide the fi­nal, de­fin­i­tive ev­i­dence its can­di­date will set the mar­ket for Pompe dis­ease drugs, if all goes well. But noth­ing has, as of yet, im­plied oth­er­wise.

Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

DFC CEO Adam Boehler and Kodak CEO Jim Continenza (Kodak)

Covid-19 roundup: Cure­Vac beefs up its uni­corn IPO dreams as bil­lion­aire own­er takes this Covid-19 mR­NA play­er on a forced march to Nas­daq; Ko­dak's $765M deal is put on hold

When CureVac initially jotted down $100 million for its IPO raise a couple of weeks ago, it seemed small. The German mRNA player, after all, had jumped into a Covid-19 race that swelled the sails of Moderna and BioNTech by tens of billions. And after raising $640 million in a slate of deals, $100 million in a hot market like this seemed like a pittance in the bigger scheme of things.

Today, we got a look at a figure that probably comes closer to the game-changing number the top execs probably have in mind. Selling 15.3 million shares at the high end of their $14 to $16 range would net a $243 million bounty. Majority owner Dietmar Hopp is putting in another €100 million, bringing the total to around $350 million. And what are the chances they want to do even better than that?

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Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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Warren Huff, Reata CEO

Rea­ta sug­gests Friedre­ich's atax­ia pro­gram could be de­layed, send­ing stock plung­ing

Reata Pharmaceuticals $RETA made waves last October when its drug omaveloxolone produced positive trial results in treating a rare neurological disorder, but the candidate’s path forward became much murkier Monday.

In a report of quarterly earnings, the biotech divulged that the FDA is considering delaying omaveloxolone’s NDA pending completion of a second trial. That could push back approval by at least a year given that the target population, individuals with Friedreich’s ataxia, is limited and progression of the hard-to-treat illness is notoriously slow. The Covid-19 pandemic would also hinder Reata’s ability to complete an additional trial.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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In­novent and Eli Lil­ly chal­lenge Mer­ck­'s mega-block­buster Keytru­da in non-small cell lung can­cer field

China-based Innovent Biologics and its multinational ally Eli Lilly shared Phase III evidence that their PD-1 inhibitor combo can delay the progression of nonsquamous non-small cell lung cancer.

But the drugmakers will face stiff competition in China from Merck’s Keytruda, the ruling PD-1 which is already approved to treat both squamous and nonsquamous NSCLC and boasts positive overall survival rates.

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Anap­tys­Bio's etokimab pro­vides more dis­ap­point­ing re­sults, rais­ing ques­tions about com­pound's fu­ture

The lead program for AnaptysBio’s in-house pipeline has hit another setback.

Etokimab, an IL-33 inhibitor, did not achieve statistically significant improvement in a Phase II trial for patients suffering from chronic rhinosinusitis with nasal polyps. Researchers measured the individuals’ bilateral nasal polyps score and sino-nasal outcome test, finding that neither improved upon a placebo after both four- and eight-week time markers, though they did demonstrate improvement over baseline levels of the examinations.

Brian Stuglik, Verastem CEO

The du­velis­ib hot pota­to is tossed to a new own­er as Ve­rastem looks to re­or­ga­nize around the pipeline

When Infinity put up duvelisib for a no-money-down instant deal, the biotech was looking for a quick exit from a clinical disaster. AbbVie had walked away from their alliance after looking at how the data stacked up in a crowded field.

And while it was approvable, it wasn’t looking pretty to anyone who thought in commercial terms.

One Big Pharma’s trash, though, was seen as a biotech treasure as a deeply troubled Verastem stepped up to grab the PI3K-delta/gamma — promising to run it across the goal lines at the FDA. And they did just that, only with little to show for it.