In a come­back, Por­to­la scores FDA ac­cel­er­at­ed ap­proval for once-re­ject­ed An­dexxa

Some­how stay­ing on the good side of the FDA, Por­to­la $PT­LA has scored an elu­sive reg­u­la­to­ry win for their once-re­ject­ed drug an­dex­anet al­fa.

The an­ti-an­ti­co­ag­u­lant — to be mar­ket­ed as An­dexxa — won the OK via the ac­cel­er­at­ed ap­proval path­way, mean­ing that Por­to­la will have to run a post-mar­ket­ing tri­al to demon­strate an abil­i­ty to con­trol bleed­ing in pa­tients.  The ap­proval was based on da­ta from two Phase III stud­ies, con­duct­ed among healthy vol­un­teers, that showed An­dexxa could re­verse an­ti­co­ag­u­lant ac­tiv­i­ty of the Fac­tor Xa in­hibitors Xarel­to (ri­varox­a­ban) and Eliquis (apix­a­ban). The drug al­so comes with a black box warn­ing and is in­di­cat­ed for use when re­ver­sal of an­ti­co­ag­u­la­tion is need­ed due to life-threat­en­ing or un­con­trolled bleed­ing.

Bill Lis

Por­to­la has fought long and hard for this win. It first sub­mit­ted an NDA for An­dexxa late 2015, on­ly to be caught off guard with an FDA re­jec­tion. At the time, the South San Fran­cis­co biotech said the agency was look­ing for more in­for­ma­tion on man­u­fac­tur­ing and how the an­ti­dote works in oth­er blood thin­ning drugs, such as edox­a­ban (Savaysa) and enoxa­parin (Lovenox).

The team filed again in Au­gust 2017, ex­act­ly a year af­ter it got the com­plete re­sponse let­ter. Reg­u­la­tors have since pushed back the ac­tion date from Feb­ru­ary to May 4, and Por­to­la hint­ed in a re­cent earn­ings call that there could be fur­ther de­lays as the FDA re­quest­ed fur­ther da­ta, send­ing stock down. But noth­ing came out of that warn­ing, and Por­to­la spread word about the OK late Thurs­day — right be­fore the orig­i­nal ac­tion date.

A spokesper­son told me that the CRL Por­to­la re­ceived in 2016 “fo­cused al­most sole­ly on man­u­fac­tur­ing con­cerns, which Por­to­la ad­dressed.”

“An­dexxa is a break­through prod­uct, and as such, clin­i­cal was much faster than man­u­fac­tur­ing,” they wrote. “In oth­er words, at that time, man­u­fac­tur­ing had to catch up with clin­i­cal.”

In a lengthy press re­lease, Por­to­la did not elab­o­rate on how it re­solved the FDA’s pre­vi­ous con­cerns, choos­ing in­stead to high­light the mar­ket po­ten­tial of its re­ver­sal agent in an era when Fac­tor Xa in­hibitors have be­come in­creas­ing pop­u­lar. One of them is Por­to­la’s own Bevyxxa, whose ap­proval raised some eye­brows since it bare­ly failed a piv­otal tri­al. Eu­ro­pean reg­u­la­tors gave it a thumbs down.

“We are proud that An­dexxa is a first-in-class med­i­cine dis­cov­ered in our labs,” said CEO Bill Lis. “We re­main com­mit­ted to our sci­en­tif­ic lead­er­ship in the fields of throm­bo­sis and hema­to­log­ic can­cers.”

The com­pa­ny now plans to launch an ear­ly sup­ply pro­gram of “Gen­er­a­tion 1” An­dexxa in June, with broad­er com­mer­cial launch an­tic­i­pat­ed in ear­ly 2019, as­sum­ing the FDA green­lights the man­u­fac­tur­ing process of its Gen­er­a­tion 2 prod­uct.

Mean­while, the post-mar­ket­ing tri­al is slat­ed to be­gin in 2019 and be re­port­ed in 2023.

Por­to­la shares edged up 3% in pre-mar­ket trad­ing. Ac­cord­ing to Eval­u­atePhar­maAn­dexxa car­ries a 2022 sell­side rev­enue fore­cast of $749m.

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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The home run count: The $100M+ mega-round boom in biotech in­spired a $6.7B feed­ing fren­zy — so far this year

Over the last 6 months there’s been a blizzard of money piling up drifts of the green stuff through the biotech landscape. And the forecast calls for more cash windfalls ahead.

Even as a global pandemic has killed more than half a million people, blighted economies and divided nations over the proper response, it’s also helped ignite an unprecedented burst of big-time investing. And not just in Covid-19 deals, as we’ve looked at before.

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For­bion spot­lights late-stage plays, carves out new €250M growth fund

Having staked its rep on picking out a mix of biotech investment opportunities across the “build,” “enable,” “growth” continuum, Forbion is launching its first fund dedicated to late-stage opportunities.

Forbion Growth Opportunities Fund’s first close brought in €185 million ($208 million). Existing investors Pantheon, KfW Capital and the European Investment Fund came on board, joined by new backers Eli Lilly, Horizon Therapeutics, Belgian Growth Fund and New Waves Investments.

Mer­ck ex­pands scope of Zymeworks an­ti­body al­liance, adding close to $900M in mile­stones

Nearly a decade after first partnering with Merck, Vancouver-based biotech Zymeworks has expanded its collaboration with the pharma giant once again.

Zymeworks re-upped with Merck in a new licensing agreement, granting the New Jersey pharma giant the right to develop up to 3 additional multispecific antibody candidates. In exchange, the biotech will receive an undisclosed upfront payment — Merck is always loath to discuss cash terms — and nearly $900 million in combined regulatory ($411 million) and commercial ($480 million) milestones.

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UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Nello Mainolfi (Kymera via YouTube)

Out to re­vive R&D, a resur­gent Sanofi pays $150M cash to part­ner up with a pi­o­neer­ing pro­tein degra­da­tion play­er

Frank Nestle was appointed Sanofi’s global head of immunology and inflammation research therapeutic area just days before dupilumab, the blockbuster-to-be IL-4 antibody, would be accepted for priority review. After four years of consolidating immunology expertise from multiple corners of the Sanofi family and recruiting new talents to build the discovery engine, he’s set eyes on a Phase I-ready program that he believes can grow into a Dupixent-sized franchise.

Atul Deshpande, Harbour BioMed chief strategy officer & head, US operations (Harbour BioMed)

An­oth­er biotech IPO set-up? Multi­na­tion­al biotech leaps from round to round, scoop­ing up cash at a blis­ter­ing pace

A short four months after announcing a $75 million haul in Series B+ fundraising, the multinational biotech Harbour BioMed pulled in another round of investments and eclipsed the nine-digit mark in the process.

Harbour completed its Series C financing, the company announced Thursday morning, raising $102.8 million and bringing its total investment sum to over $300 million since its founding in late 2016. The biotech plans to use the money to transition early-stage candidates from the discovery phase, fund candidates already in the clinic, and prep late-stage candidates for commercialization.

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Covid-19 roundup: CDC de­bat­ing who should get first avail­able vac­cines; EU in Gilead talks af­ter US gob­bled first remde­sivir dos­es

The federal government has now spent billions of dollars accelerating the development of a Covid-19 vaccine, and yet they’ve remained hush-hush on who, precisely, would actually get inoculated once the first doses are approved and available. Internally, though, they have been debating it.

The CDC and an advisory committee of outside health experts have been working since April to devise a ranking system that would determine who receives a vaccine and when, The New York Times reported. The question of who is first in line for inoculation is important because no matter how many doses developers can make or how quickly they can make them, doses will still come out in batches; 300 million inoculations will not appear overnight.

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Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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