Sage, Bio­gen head to FDA with $3.1B de­pres­sion drug; Sana puts down $50M to tap Beam's CRISPR tech

Weeks af­ter an­nounc­ing they would drop a pair of stud­ies deemed un­nec­es­sary to file the de­pres­sion drug zu­ra­nolone for ap­proval, Sage and Bio­gen are go­ing for it.

Fol­low­ing a pre-NDA meet­ing with the FDA, the part­ners say they plan to sub­mit an NDA in the sec­ond half of 2022 for ma­jor de­pres­sive dis­or­der, with a sec­ond for post­par­tum de­pres­sion to fol­low in 2023.

“We have iden­ti­fied what we be­lieve is the most ef­fi­cient path for­ward for an FDA fil­ing and po­ten­tial ap­proval,” Sage CEO Bar­ry Greene said in a state­ment.

They will be car­ry­ing on that path da­ta from com­plet­ed stud­ies in the LAND­SCAPE and NEST pro­grams, plus re­sults from on­go­ing clin­i­cal and phar­ma­col­o­gy stud­ies — al­though they didn’t spec­i­fy which ones. It could in­clude the CORAL study, which the com­pa­nies not­ed is ful­ly en­rolled with topline da­ta ex­pect­ed in ear­ly 2022.

De­spite an ear­ly flop with the MOUN­TAIN study and mixed re­sults in the more re­cent WA­TER­FALL read­out, Sage and Bio­gen (which de­vised a $3.1 bil­lion deal to team up on the drug) have re­mained con­fi­dent about zu­ra­nolone.

But then again, Bio­gen has nev­er shied away from con­tro­ver­sial ther­a­pies, ex­em­pli­fied by Aduhelm and more re­cent­ly the ALS drug tofersen.

“The ef­fi­ca­cy and safe­ty da­ta planned for FDA sub­mis­sion sup­port our vi­sion of zu­ra­nolone be­ing an as-need­ed, two-week, once-dai­ly treat­ment op­tion for MDD and PPD that pro­duces rapid re­lief from symp­toms with­in days,” Al San­drock, Bio­gen’s R&D chief, said in a state­ment. — Am­ber Tong

Sana puts down $50M to tap Beam’s CRISPR tech

The cell ther­a­py ex­perts at Sana have picked up a new tool to tin­ker with their cells.

Pay­ing Beam Ther­a­peu­tics $50 mil­lion up­front grants Sana ac­cess to its CRISPR Cas12b nu­cle­ase sys­tem, which en­ables sci­en­tists to knock out and knock in genes in cer­tain cell types — ex­treme­ly use­ful for the ex vi­vo al­lo­gene­ic T cell and stem cell-de­rived pro­grams at Sana.

“Gene edit­ing tech­nol­o­gy is a key com­po­nent in de­vel­op­ing en­gi­neered cells as med­i­cines,” Sana CEO Steve Harr said. “The speci­fici­ty and ef­fi­cien­cy of Cas12b make it ap­peal­ing for Sana’s al­lo­gene­ic T cell as well as gene-edit­ed pluripo­tent stem cell pro­grams.”

The first IND in­volv­ing this tech­nol­o­gy could be filed as ear­ly as next year, he added, with many more to come. — Am­ber Tong

Boehringer In­gel­heim ex­er­cis­es IP op­tion to de­vel­op CF cell ther­a­py

Ger­man phar­ma Boehringer In­gel­heim an­nounced to­day that it has ex­er­cised op­tions on in­tel­lec­tu­al prop­er­ty to progress and ac­cel­er­ate de­vel­op­ment of a po­ten­tial treat­ment for cys­tic fi­bro­sis, or CF.

Boehringer has been work­ing with the IP Group, the UK Cys­tic Fi­bro­sis Gene Ther­a­py Con­sor­tium and Ox­ford Bio­med­ica on a mul­ti-field part­ner­ship since 2018. Now the IP Group, act­ing on be­half of GTC host uni­ver­si­ties (Im­pe­r­i­al Col­lege in Lon­don and the Uni­ver­si­ties of Ox­ford and Ed­in­burgh), will grant ex­clu­sive glob­al rights to Boehringer to de­vel­op, man­u­fac­ture, reg­is­ter and com­mer­cial­ize a lentivi­ral vec­tor-based gene ther­a­py for the treat­ment of cys­tic fi­bro­sis. Ox­ford Bio­med­ica will be chip­ping in its man­u­fac­tur­ing knowhow.

This deal be­tween acad­e­mia, biotech and ven­ture cap­i­tal has cen­tered around BI-3720931, an in­hala­tion-based lentivi­ral vec­tor which would ide­al­ly in­tro­duce a healthy CFTR gene in­to rel­e­vant cells.

Per the agree­ment from Au­gust 2018, Boehringer In­gel­heim will pay IP Group an op­tion ex­er­cise fee and mile­stone pay­ments, as well as roy­al­ties. Ox­ford Bio­med­ica will re­ceive an op­tion ex­er­cise fee of $4.8 mil­lion and will be en­ti­tled to pay­ments in an ag­gre­gate amount of up to $38 mil­lion up­on reach­ing var­i­ous mile­stones, plus roy­al­ties. – Paul Schloess­er

Bris­tol My­ers li­cens­es fifth Drag­on­fly drug

Cel­gene was the first com­pa­ny to jump on Drag­on­fly’s TriN­KET plat­form, but the new boss­es at Bris­tol My­ers Squibb have li­censed more and more of the can­di­dates. Which leads us to Mon­day, when Drag­on­fly an­nounced that Bris­tol My­ers li­censed its fifth drug can­di­date from the plat­form.

That brings the to­tal drug can­di­dates li­censed by Bris­tol My­ers to six — in­clud­ing Drag­on­fly’s IL-12 cy­tokine BMS-986415.

Drag­on­fly al­so an­nounced Mon­day that the first pa­tients have been dosed in Phase I clin­i­cal tri­als of in­ves­ti­ga­tion­al im­munother­a­pies CC-96191 and CC-92328, both of which are li­censed to Bris­tol My­ers.

The im­munother­a­pies are be­ing test­ed for re­lapsed or re­frac­to­ry acute myeloid leukemia and re­lapsed and/or re­frac­to­ry mul­ti­ple myelo­ma, re­spec­tive­ly. These are the sec­ond and third TriN­KET can­di­dates in the clin­ic.

“We be­lieve this re­cent opt-in de­ci­sion by Bris­tol My­ers Squibb fur­ther val­i­dates our drug dis­cov­ery plat­form,” said Drag­on­fly CEO Bill Haney. – Paul Schloess­er

Clo­vis On­col­o­gy en­lists ITM for long-term ra­dioiso­tope sup­ply

Ra­dio­phar­ma new­bie Clo­vis On­col­o­gy has turned to a more ex­pe­ri­enced play­er in the space for a clin­i­cal sup­ply agree­ment, the com­pa­nies an­nounced Tues­day.

The deal pro­vides Clo­vis with ITM’s pu­ri­fied ra­dioiso­tope Lutetium-177 to use in de­vel­op­ing FAP-2286, Clo­vis’ fi­brob­last ac­ti­va­tion pro­tein (FAP)-tar­get­ing ther­a­peu­tic can­di­date.

FAP-2286 is cur­rent­ly be­ing in­ves­ti­gat­ed in a Phase I/II study for pa­tients with ad­vanced sol­id tu­mors. The deal is ini­tial­ly for five years, and no oth­er de­tails of the arrange­ment were re­leased.

“Clo­vis On­col­o­gy is com­mit­ted to ad­vanc­ing FAP-2286’s clin­i­cal de­vel­op­ment pro­gram … A crit­i­cal el­e­ment to ad­vance this pro­gram is en­sur­ing long-term sup­ply of ra­dioiso­topes, and this agree­ment al­lows us to achieve that goal,” said Clo­vis pres­i­dent and CEO Patrick Ma­haffy. – Paul Schloess­er

D&D Phar­mat­e­ch bags $51M to fu­el di­a­betes, NAFLD tri­als

South Ko­re­an biotech D&D Phar­mat­e­ch re­vealed Tues­day that it closed a $51 mil­lion Se­ries C round led by Prax­is Cap­i­tal Part­ners.

Oth­er in­vestors in­clude DS As­set Man­age­ment, Ku­dos Ven­tures and Ko­rea In­vest­ment & Se­cu­ri­ties. The new round comes af­ter a $137.1 mil­lion Se­ries B round was com­plet­ed in Au­gust 2019. D&D is plan­ning an IPO in Ko­rea next year.

The funds will be used to ad­vance the dual ag­o­nist can­di­date DD01 in­to a Phase II tri­al for obese pa­tients with both di­a­betes and non­al­co­holic fat­ty liv­er dis­ease (NAFLD), as well as sup­port Phase II stud­ies of pa­tients with Parkin­son’s and Alzheimer’s dis­ease, and pa­tients with fi­brot­ic dis­or­ders.

“We are grate­ful for the on­go­ing sup­port and con­fi­dence of our in­vestors and ex­cit­ed to have had Prax­is Cap­i­tal lead this Se­ries C fi­nanc­ing,” said D&D Phar­mat­e­ch CEO Yoo-Seok Hong in a state­ment. “We look for­ward to the on­go­ing ad­vance­ment of our clin­i­cal can­di­dates.” – Paul Schloess­er

Or­biMed joins $33M round for pet gene ther­a­py start­up

Pet gene ther­a­py biotech Scout Bio an­nounced the clos­ing of a Se­ries B2 fi­nanc­ing, which put $33 mil­lion in the bank.

This fi­nanc­ing round in­clud­ed new in­vestor Or­biMed and pre­vi­ous Se­ries B1 in­vestors Dig­i­tal­is Ven­tures, Fra­zier, River­Vest Ven­ture, among oth­ers.

Pro­ceeds from the fi­nanc­ing will ad­vance clin­i­cal de­vel­op­ment of Scout Bio’s prod­uct can­di­dates tar­get­ing di­a­betes, fe­line pain as­so­ci­at­ed with os­teoarthri­tis and CKD-as­so­ci­at­ed ane­mia, in ad­di­tion to re­search on oth­er in­di­ca­tions.

“We are pleased to wel­come the val­i­dat­ing sup­port of world-class in­vestors who share our ex­cite­ment for in­no­va­tion in vet­eri­nary med­i­cine,” said CEO Mark Hef­fer­nan. “Scout Bio is on tar­get to de­liv­er on our plat­form over the next twelve months, mak­ing sin­gle-shot ther­a­pies ex­press­ing val­i­dat­ed pro­tein ther­a­peu­tics a re­al­i­ty.” – Paul Schloess­er

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Re­searchers move clos­er to de­ci­pher­ing blood clots from As­traZeneca, J&J's Covid-19 vac­cines

Researchers may be nearing an answer for the mysterious and life-threatening blood clots that appeared on very rare occasions in people who received the J&J or AstraZeneca Covid-19 vaccine.

The new work builds on an early hypothesis researchers in Norway put forward last spring, when the cases first cropped up. They proposed the events were similar to blood clots that can occur in a small subset of patients who receive heparin, one of the most commonly used blood thinners.