Mallinck­rodt up­dates spin-off plans; FDA ex­pands Vray­lar ap­proval for Al­ler­gan; Ju­ve­nes­cence spawns Sou­vien Ther­a­peu­tics

→ UK-based bio­phar­ma­ceu­ti­cal com­pa­ny Mallinck­rodt $MNK — the mak­er of the con­tro­ver­sial drug Ac­thar — has up­dat­ed its plans for its new spin-off com­pa­ny, first an­nounced last year. The new plan is to di­vide the com­pa­ny in­to two in­de­pen­dent pub­licly trad­ed com­pa­nies: one en­cap­su­lat­ing the spe­cial­ty gener­ics/ac­tive phar­ma­ceu­ti­cal in­gre­di­ents side of the busi­ness, while the oth­er will en­com­pass the spe­cial­ty brand­ed phar­ma­ceu­ti­cals busi­ness.

The spe­cial­ty gener­ics/API busi­ness will re­tain the Mallinck­rodt name, be list­ed on the NYSE and as­sume the MNK tick­er sym­bol, while the spe­cial­ty brands com­pa­ny will con­tin­ue to be list­ed on the NYSE, be re­named Sono­rant Ther­a­peu­tics, and adopt SRTX as its sym­bol.

The com­plet­ed spin-off is pro­ject­ed to oc­cur in the sec­ond half of 2019, and the com­pa­ny ex­pects up to $300 mil­lion in fresh debt to be raised.

→ The FDA has ex­pand­ed the use of Al­ler­gan’s $AGN Vray­lar to treat de­pres­sive episodes of bipo­lar I dis­or­der. Un­til now, Vray­lar is an ap­proved treat­ment in the US for schiz­o­phre­nia in adults and for man­ic or mixed episodes as­so­ci­at­ed with bipo­lar I dis­or­der. The drug gen­er­at­ed more than a 70% year-over-year sales jump in the first quar­ter. It is es­ti­mat­ed that near­ly 11 mil­lion adults in the US live with bipo­lar dis­or­der.

Lau­ra Sepp-Loren­zi­no Linkedin

Ju­ve­nes­cence’s new ven­ture Sou­vien Ther­a­peu­tics is cre­at­ing in­no­v­a­tive med­i­cines to ad­dress neu­rode­gen­er­a­tive dis­eases. Sou­vien was found­ed “on the pi­o­neer­ing re­search of pro­fes­sor Li-Huei Tsai, di­rec­tor of The Pi­cow­er In­sti­tute for Learn­ing and Mem­o­ry at MIT, and as­so­ciate pro­fes­sor Stephen Hag­gar­ty, the di­rec­tor of the chem­i­cal neu­ro­bi­ol­o­gy lab­o­ra­to­ry at Har­vard Med­ical School/Mass­a­chu­setts Gen­er­al Hos­pi­tal, on the epi­ge­net­ic un­der­pin­nings of neu­rode­gen­er­a­tion.”

Li-Huei Tsai Pi­cow­er In­sti­tute

“We have col­lect­ed a sub­stan­tial body of da­ta im­pli­cat­ing a crit­i­cal epi­ge­net­ic reg­u­la­tor of cel­lu­lar ag­ing in neu­rode­gen­er­a­tion” said Tsai in a state­ment.

Stephen Hag­gar­ty Hag­gar­ty Lab

CEO of Ju­ve­nes­cence, Greg Bai­ley, com­ment­ed: “De­men­tia and neu­rode­gen­er­a­tive con­di­tions are a tremen­dous bur­den on our health­care sys­tem and econ­o­my, and we are ex­cit­ed by the po­ten­tial for Sou­vien to have an im­pact on this un­met clin­i­cal need.”

→ CRISPR/Cas9 tech­nol­o­gy user In­tel­lia Ther­a­peu­tics $NT­LA wel­comed Lau­ra Sepp-Loren­zi­no as its new ex­ec­u­tive vice pres­i­dent and CSO. Sepp-Loren­zi­no joins the com­pa­ny af­ter a stint at Ver­tex Phar­ma­ceu­ti­cals $VRTX as vice pres­i­dent, head of nu­cle­ic acid ther­a­pies, re­search lead­er­ship and a mem­ber of the ex­ter­nal in­no­va­tion team. Pri­or to Ver­tex, Sepp-Loren­zi­no de­vel­oped and im­ple­ment­ed the he­pat­ic in­fec­tious dis­ease strat­e­gy at Al­ny­lam Phar­ma­ceu­ti­cals $AL­NY. Be­fore that, she spent 14 years at Mer­ck $MRK ad­vanc­ing RNAi as a nov­el ther­a­peu­tic modal­i­ty. 

→ It’s time for Ama­ran­tus Bio­science {$AMBS } to go the strate­gic al­ter­na­tives route. The pen­ny stock biotech has brought in Evo­lu­tion Ven­ture Part­ners to check out po­ten­tial busi­ness op­por­tu­ni­ties and strate­gic al­ter­na­tives in the hemp sec­tor. “The en­gage­ment is for a pe­ri­od of six months, dur­ing which time the com­pa­ny will con­tin­ue to ex­e­cute on its bio­phar­ma­ceu­ti­cal hold­ing com­pa­ny fi­nanc­ing and spin­off mod­el,” the com­pa­ny said.

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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In­cyte ponies up $12M to set­tle char­i­ty foun­da­tion kick­back claims; US ex­er­cis­es op­tion for more dos­es of mon­key­pox vac­cine

One in a string of lawsuits targeting copay charity foundations, the DOJ has been hunting drugmaker Incyte for what prosecutors alleged was a kickback scheme to court patients. Now, Incyte is clearing its name.

Incyte will shell out $12.6 million to settle claims it funneled funds through a charity foundation to cover federal copays for patients taking its JAK inhibitor Jakafi, the DOJ said this week.

Angela Merkel (AP Photo/Michael Sohn)

Covid-19 roundup: Pfiz­er sub­mits vac­cine for full ap­proval; Merkel op­pos­es Biden pro­pos­al to sus­pend IP for vac­cines

Pfizer and BioNTech said Friday that they’ve submitted a biologics license application to the FDA for full approval of their mRNA vaccine for those over the age of 16.

How long it will take the FDA to decide on the BLA will be set once it’s been formally accepted by the agency.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, previously told Endpoints News that the review of the BLA should take between three and four months, but it may be even faster than that.

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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An­oth­er failed tri­al for Or­p­hazyme's 'pipeline-in-a-pro­duc­t' leaves shad­ow on drug's fu­ture

The tumultuous ride for Orphazyme continued on Friday as the company announced that a pivotal trial for its lead drug arimoclomol failed yet again, this time in the treatment of ALS, seeding doubt in a drug that had recently been cleared by the FDA for priority review. The latest failure casts a darker shadow on the upcoming decision despite Orphazyme’s upbeat outlook.

In a statement, the Danish biotech announced that the drug did not meet its primary or secondary endpoints evaluating function and survival. But the company has not announced any data surrounding the failure, instead saying that it will publish the complete results later this year.

UP­DAT­ED: EMA safe­ty com­mit­tee seeks more in­fo on heart in­flam­ma­tion fol­low­ing Pfiz­er Covid-19 vac­cine

The European Medicines Agency’s safety committee said Friday that it’s aware of cases of inflammation of the heart muscle and inflammation of the membrane around the heart, mainly reported following vaccination with Pfizer’s Covid-19 vaccine, known in Europe as Comirnaty.

“There is no indication that these cases are due to the vaccine,” the EMA’s Pharmacovigilance Risk Assessment Committee said.

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As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

AstraZeneca’s closely-watched POSEIDON study continues to be the rare bright spot in its push for an in-house PD-L1/CTLA-4 combo.

Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

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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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