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

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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A Mer­ck part­ner is sucked in­to the fi­nan­cial quag­mire as key lender calls in a note

Another biotech standing on shaky financial legs has fallen victim to the bears.

Merck partner 4D Pharma has reported that a key lender, Oxford Finance, shoved the UK company into administration after calling in a $14 million loan they couldn’t immediately make good on. Trading in their stock was halted with a market cap that had fallen to a mere £30 million.

“Despite the very difficult prevailing market conditions,” 4D reported on Friday, the biotech had been making progress on finding some new financing and turned to Oxford with an alternative late on Thursday and then again Friday morning.

Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.

Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

Chris Anzalone, Arrowhead CEO

Take­da, Ar­row­head spot­light da­ta from small tri­al show­ing RNAi works in a rare liv­er con­di­tion

Almost two years after Takeda wagered $300 million cash to partner with Arrowhead on an RNAi therapy for a rare disease, the companies are spelling out Phase II data that they believe put them one step closer to their big dreams.

In a small, open label study involving only 16 patients who had liver disease associated with alpha-1 antitrypsin deficiency (AATD), Arrowhead’s candidate — fazirsiran, previously ARO-AAT — spurred substantial reductions in accumulated mutant AAT protein in the liver, a hallmark of the condition. Investigators also tracked improvements in symptoms, with seven out of 12 who received the high, 200 mg dose seeing regression of liver fibrosis.

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Mi­to­chon­dria biotech re­ceives Morn­ing­side buy­out of­fer af­ter PhII fail; On­col­o­gy mi­cro­cap with­draws IPO plans

Not even two months after reporting a Phase II fail for its lead drug candidate, Stealth BioTherapeutics is now being eyed for a potential acquisition, with one firm wanting to take it private.

Stealth reported Monday that Morningside Venture Investments sent Stealth’s board of directors a non-binding preliminary proposal to acquire all outstanding shares not yet owned by Morningside for 2.6 cents per share and 31.3 cents per ADS.