PhI­II de­tails boost Acor­da as ex­ecs rush to FDA with the clock tick­ing on a time bomb

Ron Co­hen, Acor­da

Shares of Acor­da got a boost af­ter the mar­ket closed on Mon­day as in­vestors got a chance to look over the de­tailed Phase III da­ta for its in­haled lev­odopa drug CVT-301. And while we al­ready knew that the top-line da­ta on the pri­ma­ry end­point for Parkin­son’s dis­ease were pos­i­tive, it seemed to help that this late-stage ther­a­py ap­pears on track to ar­rive at the FDA lat­er this month as com­pa­ny ex­ecs look to re­place its big mon­ey­mak­er, now poised to lose patent pro­tec­tion.

Fail­ure, or even the hint of a set­back here, would be cat­a­stroph­ic. So the p=0.009 val­ue for a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in mo­tor func­tion among pa­tients ex­pe­ri­enc­ing OFF times was re­as­sur­ing. The switch from OFF to ON— mark­ing pe­ri­ods of the day when the drug is work­ing— and stay­ing ON at 60 min­utes, a key sec­ondary end­point, was al­so pos­i­tive. But the third step in the sec­ondary re­view ran in­to trou­ble, with no sig­nif­i­cant change to the Uni­fied Parkin­son’s Dis­ease Rat­ing Scale — III at 20 min­utes.

An­a­lysts al­so flagged some wor­ries about the lack of sig­nif­i­cant im­prove­ment in to­tal OFF time, which could crimp its roll­out, pro­vid­ed reg­u­la­tors of­fer a green light in the first place.

Acor­da’s shares jumped 10% af­ter in post-mar­ket trad­ing.

Paul Mat­teis at Leerink of­fered a quick thumbs up:

We be­lieve the full re­sult – con­veyed at the Move­ment Dis­or­ders So­ci­ety An­nu­al Meet­ing – sup­ports our fair­ly high, 90% odds of FDA ap­proval. With­in the full da­ta we are en­cour­aged with what looks to be an ac­cept­able safe­ty pro­file as well as a low, place­bo-like dropout rate. Sec­ondary end­points al­so most­ly trend or are clear­ly in fa­vor of drug – in­clud­ing the pa­tient glob­al im­pres­sion of change – though the lack of dif­fer­ence (p-val­ue of 0.98) on to­tal OFF time may emerge as a sub­ject of dis­cus­sion as in­vestors de­bate the size of the com­mer­cial op­por­tu­ni­ty. We mod­el ~$278MM in CVT-301 sales in 2022E, and we ex­pect the prod­uct to be ap­proved in the mid­dle of next year.

Acor­da shift­ed to sur­vival mode in ear­ly April af­ter a US dis­trict court tossed four key patents on its flag­ship drug Ampyra, leav­ing it with one to stand on in­to next year. The com­pa­ny chopped 20% of its staff as it scram­bles to re­struc­ture while gam­bling that it can field new drugs in short or­der.

But Acor­da CEO Ron Co­hen left his mar­ket­ing team in place, gam­bling that an on-time ap­proval would al­low for a quick hand­off of CVT-301 as they scram­ble to pre­vent a plunge in rev­enue. Ampyra pro­vides the bulk of the com­pa­ny’s mon­ey.

Even with high odds of an ap­proval, though, biotech is known for any num­ber of sna­fus that can af­flict the progress of a drug. And this time there’s no room for er­ror as they stay fo­cused on the high wire act.

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

This is the second biotech buyout pact today, marking a brisk tempo of M&A deals in the lead-up to the big JP Morgan gathering in mid-January. It’s no surprise the acquisitions are both for cancer drugs, where Sanofi will try to make its mark while Merck beefs up a stellar oncology franchise. And bolt-ons are all the rage at the major pharma players, which you could also see in Novartis’ recent $9.7 billion MedCo buyout.

ArQule — which comes out on top after their original lead drug foundered in Phase III — highlighted early data on ‘531 at EHA from a group of 6 chronic lymphocytic leukemia patients who got the 65 mg dose. Four of them experienced a partial response — a big advance for a company that failed with earlier attempts.

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Sanofi’s big week in­cludes a promis­ing PhI­II for an or­phan dis­ease drug, with plans for a pitch to the FDA

The biopharma R&D food chain is paying off with a plan at Sanofi to pitch regulators on a new drug for an orphan disease called cold agglutinin disease.

The pharma giant ushered out a statement Tuesday morning — after it spelled out plans to radically restructure the company, abandoning cardio and diabetes research altogether — saying that their C1s inhibitor sutimlimab had cleared the pivotal study.

In­scrip­ta push­es fund­ing to $260 mil­lion as they launch genome edit­ing plat­form

Inscripta presentations can begin with the advent of agriculture. Or even further back: The emergence of man.

“We’ve come a long way, starting with natural selection,” Inscripta microbial director Nandini Krishnamurthy told a session this year at SynBioBeta, the new annual conference for the synthetic biology field.

Behind her was a slide that’s recurred in company presentations, showing from left to right across the bottom the classic evolution-of-man chart (the ‘humorous’ kind that ends on an overweight soda-drinker), a picture showing the development of corn from thin strand to bulbous Iowan, and then a squiggly protein close-up of “directed evolution.” Below that runs an arrow and a ticker of how long each takes, from 10^9 years to 1.

Re­pub­li­cans un­veil a drug price bill to ri­val the De­moc­rats — promis­ing low­er prices and more cures

Nancy Pelosi unveiled the Democrats’  drug pricing bill back in September and brought the fight straight to the industry with a proposal to empower the US government to negotiate prices for select drugs. Republicans, who decried the bill reeks of heavy-handed government intervention which will stifle innovation, now have a counterproposal they claim will result in cheaper drugs and incentivize R&D — further clouding the prospects of a bipartisan compromise that could land on Donald Trump’s desk.

Left top to right: Mark Timney, Alex Denner, Vas Narasimhan. (The Medicines Company, Getty, AP/Endpoints News)

In a play-by-play of the $9.7B Med­Co buy­out, No­var­tis ad­mits it over­paid while of­fer­ing a huge wind­fall to ex­ecs

A month into his tenure at The Medicines Company, new CEO Mark Timney reached out to then-Novartis pharma chief Paul Hudson: Any interest in a partnership?

No, Hudson told him. Not now, at least.

Ten months later, Hudson had left to run Sanofi and Novartis CEO Vas Narasimhan was paying $9.7 billion for the one-drug biotech – the largest in the string of acquisitions Narasimhan has signed since his 2017 appointment.

The deal was the product of an activist investor and his controversial partner working through nearly a year of cat-and-mouse negotiations to secure a deal with Big Pharma’s most expansionist executive. It represented a huge bet in a cardiovascular field that already saw two major busts in recent years and brought massive returns for two of the industry’s most eye-raising names.

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Chris Garabedian. Perceptive

Per­cep­tive teams up with Chris Garabe­di­an to open up a new, $210M biotech fund fo­cused on A rounds

Perceptive Advisors is one of those prolific biotech investor groups which has traditionally enjoyed zeroing in on clinical-stage investments and crossover rounds, a group that prefers more established drug development players with near-term payoff potential.

But now they’re partnering with Xontogeny chief and longtime biotech entrepreneur Chris Garabedian on a $210 million fund — with money contributed by institutional investors and family funds — to go into the launch space with their first early-stage VC fund. Dubbed the Perceptive Xontogeny Venture Fund, LP, or just PXV Fund, they plan to favor upstarts that Garabedian is fostering in his incubator. But they’ll also plan to reach outside that inner circle for more A rounds to back, with plans to dominate initial funding with $10 million to $20 million per newborn biotech.

Paul Hudson. Sanofi

New Sanofi CEO Hud­son adds next-gen can­cer drug tech to the R&D quest, buy­ing Syn­thorx for $2.5B

When Paul Hudson lays out his R&D vision for Sanofi tomorrow, he will have a new slate of interleukin therapies and a synthetic biology platform to boast about.

The French pharma giant announced early Monday that it is snagging San Diego biotech Synthorx in a $2.5 billion deal. That marks an affordable bolt-on for Sanofi but a considerable return for Synthorx backers, including Avalon, RA Capital and OrbiMed: At $68 per share, the price represents a 172% premium to Friday’s closing.

Synthorx’s take on alternative IL-2 drugs for both cancer and autoimmune disorders — enabled by a synthetic DNA base pair pioneered by Scripps professor Floyd Romesberg — “fits perfectly” with the kind of innovation that he wants at Sanofi, Hudson said.

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