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

Spe­cial re­port: Meet 20 ex­tra­or­di­nary women who are su­per­charg­ing bio­phar­ma R&D

Even though many biopharma leaders have come together in recent years to address its gender gap, the consensus is clear: We still have a long way to go.

Companies this year were 2.5 times more likely than last year to have a diversity and inclusion program in place, according to a recent BIO survey, but women are still largely absent from executive roles. Getting women to enter the industry isn’t the problem — studies show that they represent just under half of all biotech employees around the world. But climbing through the ranks can be challenging, as women still report facing stereotypes, and, unfortunately, harassment.

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Op­ti­miz­ing Oral Drug De­liv­ery us­ing Zy­dis® Oral­ly Dis­in­te­grat­ing Tablet Tech­nol­o­gy to Ad­dress Pa­tient Chal­lenges

KEY POINTS

Patients prefer oral dosing, but swallowing tablets can be a challenge for many patients.
The Zydis® orally disintegrating tablet (ODT) platform addresses challenges associated with oral dosing, expanding benefits for patients and options for healthcare providers.
A strong growth trajectory is expected for ODTs given therapeutic innovation and continued technology development.

Many patients prefer conventional tablets for the administration of medications, but some geriatric and pediatric patients and those with altered mental status and physical impairments find swallowing tablets to be difficult. Orally disintegrating tablets (ODTs), which dissolve completely without chewing or sucking, offer a patient-friendly dosage form for the administration of small-molecule drugs, peptides and proteins. With the potential for multiple sites of drug absorption, often faster onset action for the active pharmaceutical ingredient (API), and potentially greater bioavailability, ODTs are an attractive option for drug developers considering first-to-market formulations or product line extensions of existing drugs with compatible API. In this report, we look at how innovation in the industry-leading Zydis ODT platform is expanding oral formulation options and bringing benefits to patients.

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Geoffrey Porges (SVB Leerink)

The 2022 wave com­ing? Top an­a­lyst says Big Phar­ma will have more than $1T avail­able to sat­is­fy its grow­ing ap­petite for biotech M&A

All through this year you could practically feel the frustration of the biotech investor class as M&A activity continued to drag behind expectations — or desires. Buyouts of public companies provide the essential juice for keeping stocks lively, and there’s been a notable lack of juice in 2021.

So is all that about to change, big time?

SVB Leerink’s Geoffrey Porges, a longtime student of biotech M&A, thinks so. In a lengthy analysis he put out last week, Porges totted up the cash flow of the major pharmas and determined that there was a good long list of industry buyers who would have around a half trillion dollars of cash to play with in 2022. Leverage that up with added debt and you could get that deal cache to $1.6 trillion.

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Mar­ket­ingRx Matchup: How Ab­b­Vie and Bio­haven ads rank in head-to-head mi­graine chal­lenge

Are you ready to rumble? DTC brands that is. MarketingRx is launching a new monthly feature today called MarketingRx Matchup. We’re pitting two pharma brands’ DTC advertising in the same therapeutic category against each other to find out what consumers and patients really think.

Market research company Leger is handling the polling and analysis each month, and I’ll be writing up the results — along with my own take — inside MRx on the first Tuesday of the month.

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Wendy Lund, Organon chief communications officer

Q&A: Organon chief com­mu­ni­ca­tions of­fi­cer Wendy Lund talks about the Mer­ck spin­off, women’s health and why it mat­ters

One of Wendy Lund’s earliest jobs was head of marketing at Planned Parenthood. As the youngest person on its management team, she introduced them to emerging new technologies, and in return, she learned the importance of fighting for what you believe in.

Now as chief communications officer at Organon, the women’s health company recently spun off by Merck, Lund is keeping that point top of mind. That’s in part because women’s health hasn’t been a spotlight therapy area for Big Pharma in years. Several companies have spun off, sold or at least considered selling women’s health assets to focus on “core” products.

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Mar­ket­ingRx roundup: Pfiz­er re-ups pneu­mo­nia ads as Mer­ck threat looms; Re­al Chem­istry founder CEO Jim Weiss steps back

Every autumn, leaves fall from the trees and people start holiday shopping – and for the last few years Pfizer debuts a new “Know Pneumonia” awareness TV ad. This year the commercial, launched a week ago, features different people who talk about why they got vaccinated against pneumococcal pneumonia. Actors portray a young female firefighter with asthma, a mechanic with heart disease and an older woman with her grandchild. A Pfizer spokesperson declined comment on the latest iteration of the long-running campaign.

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Mer­ck pumps the brakes on two more PhI­II tri­als for its lead an­ti-HIV drug

After trial investigators flagged a drop in immune cell counts that an external committee determined was related to treatment last month, Merck has been pausing HIV-related Phase II and III trials ever since.

On Monday, the biopharma company announced it’s pausing enrollment in two of its Phase III trials evaluating its leading anti-HIV drug candidate, which is the once-monthly, oral islatravir.

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vTv Ther­a­peu­tics cuts 65% of em­ploy­ees in shake­up; Freenome bags $300M in Se­ries D to ex­pand mul­ti­omics plat­form

vTv Therapeutics has decided to chop down its workforce by almost two-thirds.

The High Point, NC biotech announced Monday that it would pivot and now prioritize its lead program, the oral glucokinase activator TTP399, as it gears up for Phase III pivotal trials.

The drug was granted breakthrough therapy designation after showing a 40% reduction in hypoglycemic episodes compared to placebo, and back in October vTv announced positive results in a study showing no increased risk for ketoacidosis, a severe complication of diabetes.

Covid-19 roundup: Plant-based shot proves safe, 71% ef­fi­ca­cious in PhI­II; Bourla ex­pects an­tivi­ral to launch this month

Quebec-based Medicago and its adjuvant partner GlaxoSmithKline said Tuesday that their plant-based Covid-19 vaccine candidate proved to be 71% efficacious against all variants of SARS-CoV-2 in a Phase III trial of more than 24,000 adults in Canada, the US, UK, Mexico, Argentina and Brazil.

In addition to showing 75% efficacy against the Delta variant specifically, the companies also said the vaccine proved to be generally safe, with no serious adverse events reported and reactogenicity generally being mild to moderate. The results mean that a regulatory submission will be filed with Health Canada imminently, they said.