Sev­en years af­ter li­cens­ing BTK in­hibitor from Bio­gen, be­lea­guered pen­ny­s­tock biotech part­ner con­cedes de­feat

A BTK in­hibitor Bio­gen punt­ed out of its pipeline years ago has flopped its first clin­i­cal test.

Day­ton Mis­feldt

Sune­sis Phar­ma said it would not be tak­ing ve­cabru­ti­nib in­to the Phase II por­tion of its Phase Ib/II tri­al af­ter re­view­ing ini­tial da­ta that sug­gest lim­it­ed ef­fi­ca­cy even at the high­est dose. The tri­al had en­rolled adult pa­tients with re­lapsed/re­frac­to­ry chron­ic lym­pho­cyt­ic leukemia (CLL) and oth­er B-cell ma­lig­nan­cies. The safe­ty pro­file was “ex­cel­lent”; it just didn’t seem to have much of an ef­fect.

Day­ton Mis­feldt, the Bay City Cap­i­tal VC who’s been in­ter­im CEO for more than two years, summed it up this way:

One par­tial re­mis­sion was ob­served af­ter 11 treat­ment cy­cles in a CLL pa­tient treat­ed in Co­hort 5 (300 mg BID) and a num­ber of pa­tients treat­ed across the dose range ex­plored (25 mg to 500 mg BID) saw sta­ble dis­ease; how­ev­er, no oth­er re­mis­sions have been ob­served.

The Phase Ib pro­gram will con­tin­ue while Sune­sis ex­ecs de­cide on the drug’s fate. Shares of the South San Fran­cis­co com­pa­ny on the Nas­daq — a pen­ny­s­tock — plum­met­ed 43.68% to $0.35 pre-mar­ket.

For now, Sune­sis will shift its fo­cus and re­sources to a PDK-1 in­hibitor dubbed SNS-510 — a process that should be fa­mil­iar to the com­pa­ny by now.

When it went pub­lic in 2005, the lead prod­uct can­di­date was SNS-595 (vosarox­in), a cell-cy­cle in­hibitor lat­er po­si­tioned for leukemia. Reg­u­la­tors spurned their Phase III da­ta and Sune­sis end­ed up out-li­cens­ing it to De­n­o­vo Bio­phar­ma, which spe­cial­izes in find­ing ap­plic­a­ble sub­groups for drugs that have com­plet­ed tri­als with “un­sat­is­fac­to­ry ef­fi­ca­cy.” Not much came out of the sec­ond project to de­vel­op an Au­ro­ra ki­nase in­hibitor, ei­ther.

The cur­rent pipeline of two con­sists en­tire­ly of as­sets orig­i­nat­ing from a col­lab­o­ra­tion with Bio­gen, who was al­so an eq­ui­ty hold­er. Aside from ve­cabru­ti­nib, a non-co­va­lent in­hibitor of BTK that promised to help Im­bru­vi­ca-re­sis­tant pa­tients, SNS-510 tar­gets PI3K-de­pen­dent and PIP3-in­de­pen­dent path­ways.

With Take­da on board as a part­ner, an IND is in the works for the end of 2020. The cash re­serves should be enough to get them there — but like­ly not that much be­yond.

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,800+ biopharma pros reading Endpoints daily — and it's free.

Covid-19 roundup: EU backs Os­i­vax's pur­suit of a uni­ver­sal vac­cine to fight Covid-19

The race to find a vaccine for the novel coronavirus continues to heat up, as the European Commission’s pilot R&D arm taps French biotech Osivax to head up its unique approach to the research.

Aiming to develop a universal jab for the flu and Covid-19, Osivax secured around $20 million in “blended financing” from the European Innovation Council. About $3 million comes from a Covid-19 “accelerator grant” and will go toward completing Osivax’s signature flu vaccine, dubbed OVX836 and currently in Phase IIa. The rest will be included as part of Osivax’s Series B funding, which aims to launch the Phase IIb portion of the study.

GSK sets the stage for a toe-to-toe mar­ket show­down with Gilead­'s HIV cham­pi­on Tru­va­da

ViiV Healthcare and majority owner GlaxoSmithKline have cleared another important hurdle on a long-running quest to challenge Gilead’s dominance in preventative HIV treatments.

The final analysis of a new study shows the GSK subsidiary’s long-lasting injection, cabotegravir, proved 66% more effective in HIV prevention than Gilead’s breakthrough Truvada pill. And they now intend to carve away some of the blockbuster revenue that Gilead has enjoyed for years.

Adrian Gottschalk, Foghorn CEO

Mer­ck dan­gles up to $425 mil­lion to team with Flag­ship’s Foghorn Ther­a­peu­tics on drug­ging the shape of DNA

Two years after it first emerged from stealth mode, Flagship’s Foghorn Therapeutics has nabbed its first Big Pharma partner as Merck signs on to the biotech’s vision of drugging the very shape of DNA.

The deal, worth up to $425 million but with the upfront cash undisclosed, comes as Foghorn nears a pivot to a clinical stage biotech. The Cambridge-based company has added nearly 60 staffers from the 25 it had when it first emerged out of Flagship and, CEO Adrian Gottschalk said, they have finally refined the screening technology at the heart of the company, with plans to file their first IND towards the end of the year.

John Reed, Sanofi R&D chief (Endpoints News)

John Reed brings NK cells in­to Sanofi's CD38 ri­val­ry with J&J — and of­fers thumbs up for Kiadis' new fo­cus

Sanofi doesn’t just want to be a challenger to J&J’s dominant Darzalex multiple myeloma franchise. It’s looking to pioneer a new approach by pairing its own — newly approved — anti-CD38 drug with an NK cell therapy it’s just picked up.

The French pharma giant has teed up $19.7 million (€17.5 million) upfront and close to a billion dollars (€857.5 million) in milestones for a license to Kiadis Pharma’s preclinical K-NK004 program, which consists of NK cells that have been genetically engineered not to express CD38.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,800+ biopharma pros reading Endpoints daily — and it's free.

Trump and Navar­ro press again for hy­drox­y­chloro­quine. Can the FDA stay in­de­pen­dent?

Tuesday morning, economist and Trump advisor Peter Navarro walked onto the White House driveway and promptly brought a political cloud back onto the FDA.

Speaking to a White House pool reporter, Navarro said that four Detroit doctors were, based on a single disputed study, filing for the FDA to again issue an emergency authorization for hydroxychloroquine, the anti-malarial pill that President Trump hyped for months as a Covid-19 treatment over the objections of his own scientists. Then, while avoiding directly calling for the FDA to OK the drug, blasted the agency. He said its decision to pull an earlier authorization “was based on bad science” and “had a tremendously negative effect” on doctors and patients.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,800+ biopharma pros reading Endpoints daily — and it's free.

David Hallal

AlloVir tests how much an an­tivi­ral biotech can reap in a pan­dem­ic stock mar­ket

The pandemic stock market has proven fruitful for virtually any type of biotech. Now a 7-year-old cell therapy startup will see how much it can yield for a company that specializes in fighting viruses.

AlloVir, a company that until 2019 largely lived off grant money, has filed for a $100 million IPO to back its line of off-the-shelf, virus-fighting T cells. Although in normal circumstances, $100 million could be a solid return for a biotech that got its first major round of funding only last year, we’ll have to wait to see how much the company ultimately earns. As Covid-19 has sent investor money scurrying to almost anyone in drug development, every single biotech to go public this year has prized above their midpoint or upsized their offering, according to Renaissance Capital, sometimes dramatically so.

Noubar Afeyan, Flagship CEO and Tessera chairman (Victor Boyko/Getty Images)

Flag­ship ex­ecs take a les­son from na­ture to mas­ter ‘gene writ­ing,’ launch­ing a star-stud­ded biotech with big am­bi­tions to cure dis­ease

Flagship Pioneering has opened up its deep pockets to fund a biotech upstart out to revolutionize the whole gene therapy/gene editing field — before gene editing has even made it to the market. And they’ve surrounded themselves with some marquee scientists and execs who have crowded around to help shepherd the technology ahead.

The lead player here is Flagship general partner Geoff von Maltzahn, an MIT-trained synthetic biologist who set out in 2018 to do CRISPR — a widely used gene editing tool — and other rival technologies one or two better. Von Maltzahn has been working with Sana co-founder Jake Rubens, another synthetic biology player out of MIT who he describes as his “superstar,” who’s taken the CSO role.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,800+ biopharma pros reading Endpoints daily — and it's free.

Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.