For­bion leads $62.5M round for neu­ro start­up as ex-Te­va R&D chief takes con­trol; CSL Behring inks gene ther­a­py pact in im­munol­o­gy

For­bion has led a $62.5 mil­lion round for Prile­nia Ther­a­peu­tics to fund two late-stage tri­als in Hunt­ing­ton’s dis­ease and amy­otroph­ic lat­er­al scle­ro­sis. Michael Hay­den, the for­mer Te­va R&D chief who’s been serv­ing as chair­man, will now take over as CEO to over­see the pro­gram for pri­do­pi­dine, which ag­o­nizes the sig­ma-1 re­cep­tor (S1R). Morn­ing­side and Sec­toral As­set Man­age­ment al­so joined, as did Tal­is­man Cap­i­tal Part­ners and Gen­works 2.

→ Aus­tralia’s CSL Behring is team­ing up with Seat­tle Chil­dren’s Re­search In­sti­tute to de­vel­op stem cell gene ther­a­pies for rare pri­ma­ry im­mun­od­e­fi­cien­cy dis­eases, start­ing with Wiskott-Aldrich Syn­drome and X-linked Agam­ma­glob­u­line­mia. They are among 400 pri­ma­ry im­mun­od­e­fi­cien­cy dis­eases, in which the hu­man im­mune sys­tem is dys­func­tion­al. “Ex­pand­ing our gene ther­a­py port­fo­lio in­to an area of im­munol­o­gy well known to CSL ex­em­pli­fies how we are strate­gi­cal­ly grow­ing our ca­pa­bil­i­ties in this strate­gic sci­en­tif­ic plat­form and are col­lab­o­rat­ing with world class in­sti­tu­tions to ac­cess in­no­va­tion with the po­ten­tial to vast­ly im­prove pa­tients’ lives,” not­ed CSL R&D chief Bill Mez­zan­otte.

Hy­loris Phar­ma­ceu­ti­cals, a Bel­gian com­pa­ny ded­i­cat­ed to re­for­mu­lat­ing well-known drugs, is look­ing to tap the pub­lic mar­ket by list­ing on Eu­ronext Brus­sels. “The IPO is ex­pect­ed to pro­vide us with the re­sources need­ed to fi­nance the de­vel­op­ment of our ex­ist­ing port­fo­lio of prod­uct can­di­dates, as well as es­tab­lish a com­mer­cial in­fra­struc­ture in the Unit­ed States for our IV Car­dio­vas­cu­lar port­fo­lio (ex­cl. So­talol IV, which is com­mer­cial­ized through a part­ner), where we will fo­cus on ad­dress­ing pre­scribers in a cost-ef­fi­cient man­ner by con­cen­trat­ing on spe­cial­ized care fa­cil­i­ties such as hos­pi­tals,” CEO Sti­jn Van Rompay said in a state­ment.

GHO Cap­i­tal has put the $1 bil­lion-plus fund it closed at the end of last year to use, buy­ing X-Chem from an af­fil­i­ate of The Car­lyle Group and Hell­man & Fried­man. The com­pa­ny spe­cial­izes in DNA-En­cod­ed Li­brary (DEL)-based dis­cov­ery ser­vices and has li­censed over 70 re­search pro­grams to bio­phar­ma part­ners over the years. Matt Clark, a co-founder and the cur­rent SVP of chem­istry, will re­place Rick Wag­n­er as CEO.

→ Dublin-based Avec­tas has en­list­ed Scott Si­mon’s lab at UC Davis to char­ac­ter­ize cells en­gi­neered on its plat­form, with the goal of in­form­ing the de­vel­op­ment of next-gen cell ther­a­pies. “A ma­jor fo­cus of our group is to un­der­stand how chem­i­cal and me­chan­i­cal forces act­ing on im­mune cells en­able them to lo­cal­ize at sites of in­flam­ma­tion,” Si­mon said in a state­ment. “The part­ner­ship with Avec­tas will help us eval­u­ate how these same forces play a role to de­liv­er­ing mR­NA and pro­teins to im­mune cells and there­by ex­tend their ther­a­peu­tic ap­pli­ca­tions.”

→ Ire­land’s Shorla Phar­ma has closed its Se­ries A at $8.3 mil­lion to ad­vance a slate of drugs for rare, or­phan and pe­di­atric can­cers. Ser­o­ba Life Sci­ences led the round, which fea­tured oth­er lo­cal in­vestors as well as fam­i­ly of­fices in Cana­da.

Chris­t­ian Anger­may­er, founder of the men­tal health and psy­che­delics biotech ATAI, is try­ing to bring the Aus­tralian biotech Bio­nom­ics and its PTSD drug back from the near-dead. Bio­nom­ic’s stock flat­lined to $0.04 af­ter the drug’s fail­ure in a Phase II tri­al, but the com­pa­ny, which has al­so part­nered with Mer­ck on Alzheimer’s, has since re­for­mu­lat­ed the drug, and Anger­may­er is bet­ting it might now work. His firm Ape­iron put down $5 mil­lion and un­der­wrote $15 mil­lion in fund­ing to put the com­pound back in­to tri­als.

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.

CEO Khurem Farooq (Gyroscope)

Hours be­fore ex­pect­ed de­but, Gy­ro­scope post­pones its IPO as 2 oth­er biotechs hold the line on their march to Nas­daq

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

In a surprising turn of events, UK-based Gyroscope Therapeutics has postponed its IPO mere hours before it was set to debut on Nasdaq.

Working on a gene therapy for wet AMD, Gyroscope was all set and ready to go public earlier this week, setting terms for a $142 million raise with a price range of $20 to $22. But in the wee hours of Friday morning, the company put out a press release saying they would delay their debut “in light of market conditions,” CEO Khurem Farooq said in a statement.

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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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