Fa­tal sep­sis at­tacks blight Acor­da’s PhI­II, trig­ger­ing new safe­ty mea­sures and tor­pe­do­ing shares

Af­ter track­ing 7 cas­es of sep­sis — with 5 deaths — in stud­ies of its late-stage Parkin­son’s drug tozadenant, Acor­da is in­creas­ing pa­tient sur­veil­lance while paus­ing en­roll­ment in its long-term safe­ty stud­ies to get a han­dle on the is­sue.

Acor­da’s shares plunged 40% on the news.

The safe­ty is­sue arose af­ter re­searchers be­came aware of the cas­es of agran­u­lo­cy­to­sis, wip­ing out the white blood cells need­ed to fight off an in­fec­tion. Now they say they will check those cell counts on a week­ly ba­sis as they study the po­ten­tial drug prob­lem with the FDA and the mon­i­tor­ing com­mit­tee.

Ron Co­hen

Acor­da CEO Ron Co­hen says they still ex­pect to re­port out Phase III safe­ty and ef­fi­ca­cy da­ta in Q1 com­ing up, but any threat to its num­ber two de­vel­op­ment pro­gram — be­hind CVT-301 — is like­ly to rat­tle its back­ers.

Leerink’s Paul Mat­teis says the sur­pris­ing news of a ma­jor safe­ty is­sue at Acor­da leaves the drug un­der a cloud.

The need for fre­quent white blood cell mon­i­tor­ing is not un­heard of for ap­proved neu­rol­o­gy prod­ucts: agran­u­lo­cy­to­sis risk is a known lim­i­ta­tion for cloza­p­ine, an ap­proved, and very ef­fec­tive atyp­i­cal an­tipsy­chot­ic. But the risk/ben­e­fit for Toz, in the con­text of an ap­par­ent mor­tal­i­ty sig­nal – and what is al­ready a crowd­ed cat­e­go­ry – is now sig­nif­i­cant­ly com­pro­mised, giv­en that the drug is not dis­ease-mod­i­fy­ing and will be tar­get­ed to­ward an al­ready frag­ile el­der­ly pop­u­la­tion.

Then, lat­er on Wednes­day, Mat­teis vir­tu­al­ly wrote it off as a los­er.

We are now very con­cerned re­gard­ing the ap­prov­abil­i­ty of Toz – even if it does work – giv­en its po­si­tion­ing as a symp­to­matic ther­a­py with a mod­er­ate lev­el of ben­e­fit. We have sig­nif­i­cant ques­tions re­gard­ing the com­mer­cial vi­a­bil­i­ty of a new symp­to­matic Parkin­son’s ther­a­py with such sig­nif­i­cant safe­ty risks.

Acor­da swooped in to buy Fin­land’s Bi­otie in ear­ly 2016 for $363 mil­lion in cash to bag the Phase III Parkin­son’s drug and add a bad­ly need­ed late-stage drug to the pipeline. Tozadenant is an oral adeno­sine A2a re­cep­tor an­tag­o­nist that’s reg­is­tered proof of con­cept da­ta for re­duc­ing “off” times among Parkin­son’s pa­tients tak­ing the stan­dard lev­odopa/car­bidopa rem­e­dy.

There’s lit­tle mar­gin for er­ror at Acor­da.

With its patents on the com­pa­ny’s cash cow Ampyra un­der at­tack, pos­si­bly open­ing the road to a gener­ic ri­val next sum­mer, the biotech has had to re-file its ap­pli­ca­tion for CVT-301. The FDA had is­sued a refuse-to-file the ap­pli­ca­tion for the drug, de­lay­ing a ther­a­py which will be need­ed to res­cue the com­pa­ny from gener­ic com­pe­ti­tion for Ampyra.

The biotech, mean­while, has moved to re­struc­ture, block a po­ten­tial hos­tile takeover at­tempt and de­flect crit­i­cism for leav­ing it­self vul­ner­a­ble to the cri­sis it’s in. If its safe­ty is­sue with tozadenant spins out of con­trol, Acor­da’s ex­ec­u­tive team will be un­der twice as much pres­sure to per­form on CVT-301.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Paul Hastings, Nkarta CEO

With no up­front pay­ment or mile­stones on the line, Nkar­ta and CRISPR join forces on CAR-NK search

Most deals in biotech come with hefty upfront payments attached, and the promise of big biobucks if a program works out. Not this one.

Nkarta has struck what CEO Paul Hastings calls a “real collaboration” with CRISPR Therapeutics to co-develop and commercialize two CAR-NK therapies, in addition to an NK+T program. The duo will split all R&D costs — and any worldwide profits — 50/50, Hastings said.

Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

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Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.

Biden ad­min­is­tra­tion backs a po­lar­iz­ing pro­pos­al to waive IP for all Covid-19 vac­cines

In a surprise U-turn, the Biden administration said Wednesday that it will support a proposal at the World Trade Organization to temporarily waive intellectual property protections on Covid-19 vaccines.

The proposal, backed by South Africa and India at the WTO, seeks to help developing countries with limited vaccine supplies. The US and Europe historically opposed the proposal, saying IP should be protected because it incentivizes new drug and vaccine development.

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