Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

Ab­b­Vie is ap­proach­ing the FDA with a new ther­a­py to po­ten­tial­ly treat Parkin­son’s dis­ease, us­ing pro­drugs of two med­ica­tions com­mon­ly used for the con­di­tion.

The Big Phar­ma sub­mit­ted its NDA for AB­BV-951, a so­lu­tion of lev­odopa and car­bidopa pro­drugs be­ing eval­u­at­ed in ad­vanced Parkin­son’s pa­tients who don’t re­spond well to oral ther­a­py, Ab­b­Vie an­nounced Fri­day morn­ing. Re­searchers are hop­ing a pos­i­tive Phase III study that reads out in late Oc­to­ber will help move things along quick­ly at the agency.

Ab­b­Vie’s the­o­ry here re­volves around the use of a con­tin­u­ous, 24-hour in­fu­sion of its ther­a­py as a unique and ef­fec­tive way to treat ad­vanced Parkin­son’s dis­ease. The lev­odopa and car­bidopa pro­drugs are de­liv­ered in small dos­es sub­cu­ta­neous­ly through a pump, and they’re de­signed to ac­ti­vate once they en­ter the blood­stream.

Much of Ab­b­Vie’s re­search thus far has cen­tered around find­ing the right con­cen­tra­tion to be able to de­liv­er the low­er lev­odopa and car­bidopa dos­es while still in­duc­ing ef­fi­ca­cious re­spons­es. Oral lev­odopa in par­tic­u­lar is one of the most com­mon­ly pre­scribed Parkin­son’s drugs, but its ef­fect wanes quick­ly and can fail to con­trol mo­tor symp­toms if the nar­row ther­a­peu­tic win­dow is missed.

In its piv­otal Phase III study, Ab­b­Vie asked pa­tients to self-re­port the times when they felt their symp­toms were well-con­trolled and when they weren’t in a spe­cial­ized di­ary. The com­pa­ny de­scribed these as “On” and “Off” times, re­spec­tive­ly. Ab­b­Vie’s pri­ma­ry end­point aimed to mea­sure the change in base­line “On” time in hours with­out in­vol­un­tary move­ments af­ter 12 weeks.

Af­ter that time, the av­er­age in­crease was 2.72 hours of “On” time for pa­tients tak­ing AB­BV-951 and 0.97 among those on stan­dard of care. Ad­di­tion­al­ly, de­creas­es of “Off” time came in at 2.75 hours in the treat­ment arm and 0.96 hours in the stan­dard of care group.

Both dif­fer­ences were sta­tis­ti­cal­ly sig­nif­i­cant, clock­ing in at re­spec­tive p-val­ues of p= 0.0083 and p=0.0054.

Ab­b­Vie has yet to pro­vide a full break­down of da­ta at a med­ical con­fer­ence, but the com­pa­ny not­ed the ad­verse event pro­file came in large­ly mild-to-mod­er­ate. Most side ef­fects had to do with the in­fu­sion site, in­clud­ing pain, red­den­ing of the skin, in­flam­ma­tion and bruis­ing. Though most were non-se­vere, a much high­er rate of pa­tients in the treat­ment arm (21.6%) dis­con­tin­ued in the study than those tak­ing stan­dard of care (1.5%).

The com­pa­ny is hop­ing this Parkin­son’s pro­gram turns out bet­ter than one of its pre­vi­ous­ly part­nered can­di­dates. Last month, Ab­b­Vie dropped its col­lab­o­ra­tion with Swe­den’s BioArc­tic around a port­fo­lio of al­pha-synu­cle­in an­ti­bod­ies, in­clud­ing one pre­vi­ous­ly ex­pect­ed to be ready for Phase II.

Should Ab­b­Vie net ap­proval here, they’d beat a cou­ple Big Phar­ma com­peti­tors to the punch. Roche and Prothena are still in Phase III on a pro­gram part­nered for $600 mil­lion, while No­var­tis inked a $1.5 bil­lion pact to get its hands on two UCB as­sets — one small mol­e­cule in­hibitor and one an­ti­body.

Paul Hudson, Sanofi CEO (Cyril Marcilhacy/Bloomberg via Getty Images)

FDA side­lines Paul Hud­son's $3.7B MS drug af­ter es­tab­lish­ing link to liv­er dam­age

One of Sanofi CEO Paul Hudson’s top picks in the pipeline — picked up in a $3.7 billion buyout 2 years ago — has just been sidelined in the US by a safety issue.

The pharma giant put out word early Thursday that the FDA has put their Phase III studies of tolebrutinib in multiple sclerosis and myasthenia gravis on partial clinical hold, halting enrollment and suspending dosing for patients who have been on the drug for less than 60 days. Patients who have completed at least 60 days of treatment can continue therapy as researchers explore a “limited” — but unspecified in Sanofi’s statement — number of cases of liver injury.

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Phar­ma re­acts to post-Roe; Drug­mak­ers beef up cy­ber de­fense; Boehringer, Roche qui­et­ly axe drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As a reminder, we are off on Monday for the Fourth of July. I hope this recap will kick off your (long) weekend well and that the rest of it will be just what you need. See you next week for a shortened edition!

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Eric Hughes, incoming Teva EVP of global R&D and CMO

Te­va chief raids Ver­tex for his new glob­al head of re­search and de­vel­op­ment

Teva CEO Kåre Schultz has found his new R&D chief and CMO in Vertex’s ranks.

The global generics giant, which has some 3,500 staffers in the R&D group, has named Eric Hughes to the top research spot in the company. He’ll be replacing Hafrun Fridriksdottir, who held the role for close to five years, on Aug. 1.

Hughes hasn’t been at Vertex for long, though. He jumped from Novartis less than a year ago, after heading the immunology, hepatology & dermatology global development unit. Before that, he completed a five-year stint as head of early clinical research for the specialty discovery medicine department in the exploratory clinical & translational research group at Bristol Myers Squibb, according to his LinkedIn profile.

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#BIO22: Man­ag­ing a biotech in tur­bu­lent times. 'There's a per­fect shit­show out there'

On Tuesday, June 14, Endpoints News EIC John Carroll sat down with a group of biotech execs to discuss the bear market for industry stocks and how they were dealing with it. Here’s the conversation, which has been lightly edited for brevity.

Martin Meeson, sponsor opening:

Thank you, John. Hello everyone. My name’s Martin Meeson, I’m the CEO of Fujifilm Diosynth. For those of you who don’t know Fujifilm Diosynth, we operate in the development of clinical and commercial product scale up, we have facilities in Europe and the US, and around about 4,000 employees. We run on average about 150 programs, so when it comes to managing in turbulent times over the last two years, we’ve had quite a lot of experience of that. Not just keeping the clinical pipelines and the commercial pipelines open, but also our response to the pandemic and the molecules that we’ve had within there. One of the phrases that I coined probably about a year ago when we were talking at JP Morgan, was I talked about managing through turbulent times. Well, it’s become the fact that we are not managing and leading through these times, we are managing in them, which is why that’s really the purpose of and the topic that we’ve got today.

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Emer Cooke, ICRMA chair (AP Photo/Geert Vanden Wijngaert)

ICM­RA to launch sev­er­al reg­u­la­to­ry pi­lot pro­grams cen­tered around man­u­fac­tur­ing in­spec­tions

As regulatory agencies look to catch up on inspections amid the Covid-19 pandemic, ICMRA is unveiling several pilot programs to address industry applications and inspections.

ICMRA, which is made up of the world’s top drug regulators, is launching multiple pilot programs, including two regulatory pilots addressing facility inspections for chemistry and manufacturing controls (CMC) and post-approval change (PAC) submission assessments and related regulatory actions.

FDA slaps warn­ing let­ter on Min­neso­ta API fa­cil­i­ty af­ter em­ploy­ee de­stroys clean­ing log

A manufacturing facility belonging to the Netherlands-based API producer Fagron Group has entered the FDA’s crosshairs after an employee destroyed a cleaning log, among other violations.

One of its plants in Saint Paul, MN received a warning letter on June 14, following an inspection last November that uncovered cross-contamination concerns.

“In your response, you provided a follow-up cleaning validation report in which you only assessed the carryover of niacin swab samples but not progesterone, which was included in your initial cleaning validation,” FDA says in the letter. “The lack of progesterone (b)(4) [commercially confidential information] is concerning considering the failing residue results you provided to investigators would yield unacceptable levels of progesterone cross-contamination.”

Susan Galbraith, AstraZeneca EVP, oncology R&D

Catch­ing up with Bris­tol My­ers and Mer­ck, As­traZeneca de­clares neoad­ju­vant win for PD-L1/chemo com­bo

When AstraZeneca started the Phase III AEGEAN trial for Imfinzi in 2018, it was, alongside several Big Pharma brethren, hoping to push the use of PD-(L)1 therapies into earlier lines of treatment. Three and a half years later, the British drugmaker has nabbed promising data in a type of lung cancer.

Topline results from an interim analysis showed that adding Imfinzi to chemotherapy before surgery spurred a “statistically significant and meaningful” improvement in pathologic complete response for patients with resectable non-small cell lung cancer compared to chemotherapy alone.

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Nassim Usman, Catalyst Biosciences CEO

Fac­ing set­backs for months and an ac­tivist at­tack, Cat­a­lyst Bio­sciences pre­pares to call it quits

After downsizing for several months, Catalyst Biosciences is getting ready to tap out.

The San Francisco biotech announced Wednesday that it would be liquidating and distributing cash back to shareholders, with total proceeds expected to reach $65 million. Catalyst intends to return the money “as soon as practicable,” the company said, as it has ceased all R&D activities, CEO Nassim Usman said in a statement.

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Amgen's taking social media followers around the globe as it introduces the many different

From Tam­pa to Mu­nich, Am­gen’s ‘Places’ cam­paign in­tro­duces its lo­ca­tions around the world

Amgen is taking social media followers around the world with its latest corporate campaign. Called “Places of Amgen,” the twice monthly posts highlight the biopharma’s different offices and sites – and the people who work there.

Each post runs on LinkedIn, Facebook and Instagram with details about the work Amgen does in that location, when it was established, comments from people who work there and other interesting facts. The most recent one about Paris, France, for example, notes that Amgen France last year signed a French association charter committed to the inclusion of LBGT+ people in the workplace.

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