As­traZeneca's di­a­betes drug grant­ed fast track des­ig­na­tion for heart fail­ure; Ver­ri­ca sends skin in­fec­tion treat­ment to FDA

A cou­ple weeks af­ter an­nounc­ing that their di­a­betes drug cut the risk of CV death or wors­en­ing of heart fail­ure by 26% in a land­mark study, As­traZeneca an­nounced that the FDA has grant­ed fast track des­ig­na­tion for Farx­i­ga in adults with heart fail­ure with re­duced ejec­tion frac­tion (HFrEF) or pre­served ejec­tion frac­tion (HF­pEF). The drug was giv­en the same des­ig­na­tion last month for re­nal fail­ure and the pre­ven­tion of re­nal death and CV in pa­tients with chron­ic kid­ney dis­ease.

→ Der­ma­tol­ogy-fo­cused Ver­ri­ca Phar­ma­ceu­ti­cals $VR­CA has sub­mit­ted an NDA for their top­i­cal ther­a­py, VP-102, for the treat­ment of mol­lus­cum con­ta­gio­sum — a high­ly con­ta­gious vi­ral skin in­fec­tion that af­fects ap­prox­i­mate­ly 6 mil­lion peo­ple, pri­mar­i­ly chil­dren, in the Unit­ed States. Back in June, the com­pa­ny re­leased pos­i­tive re­sults of the drug from its COVE-1 Phase II study for the treat­ment of ver­ru­ca vul­garis, or com­mon warts.

Wave Life Sci­ences $WVE — which en­tered in­to a meaty part­ner­ship with Take­da last year — has se­cured fast track sta­tus from the FDA for its Duchenne drug, which is en­gi­neered to di­rect­ly com­pete with Sarep­ta‘s pi­o­neer­ing Ex­ondys 51. The ther­a­py, called su­vodirsen, is cur­rent­ly be­ing test­ed in a Phase II/III study, and Wave is gear­ing up for reg­u­la­to­ry sub­mis­sions in the sec­ond half of next year.

Anavex broke out some pre­lim­i­nary da­ta from a mid-stage tri­al in­volv­ing pa­tients with Rett syn­drome, a ge­net­ic brain dis­or­der that oc­curs al­most ex­clu­sive­ly in girls. Pa­tients in the tri­al were ei­ther giv­en a 5 mg dai­ly oral liq­uid dose of the ex­per­i­men­tal drug, blar­came­sine, or place­bo. The com­pa­ny $AVXL dis­closed that in six pa­tients who were giv­en the drug, there were im­prove­ments in two sec­ondary end­points — the Rett Syn­drome Be­hav­iour Ques­tion­naire and the Clin­i­cal Glob­al Im­pres­sion — af­ter sev­en weeks of treat­ment.

Glax­o­SmithK­line has won an ex­pand­ed FDA ap­proval for Nu­calaex­tend­ing its reach to a young pa­tient pop­u­la­tion. The IL-5 drug, which was first ap­proved in 2015, can now be used to treat chil­dren as young as 6 with se­vere eosinophilic asth­ma.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

At­las rais­es new $400M fund amid spree of VC rais­es. Here’s what they’ll spend it on

You can add another few hundred million to the now Montana-sized reservoir of cash biotech VCs have raised since the WHO declared Covid-19 a pandemic.

Atlas Ventures, the prominent Kendall Square incubator, has raised $400 million for its twelfth biotech fund, their first in 3 years. After a string of mammoth new raises from other major VCs in April and May, the total pot now stands between $5 billion and $6 billion, depending on how you slice it.

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

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Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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