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.

UP­DAT­ED: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,600+ biopharma pros reading Endpoints daily — and it's free.

(Image: Associated Press)

Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,600+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,600+ biopharma pros reading Endpoints daily — and it's free.

(Image: Associated Press)

No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,600+ biopharma pros reading Endpoints daily — and it's free.

As­traZeneca gains EU nod for di­a­betes triple; Am­gen and Duke launch re­al-world PC­SK9 ob­ser­va­tion­al study

→ Weeks after winning EU approval to start marketing dapagliflozin as Forxiga, AstraZeneca has racked up another OK for a triplet combo involving the SGLT2 diabetes drug. Named Qtrilmet, the pill combines Forxiga with the DPP-4 inhibitor Onglyza (saxagliptin) and the bedrock drug metformin in a modified-release format. That 3-in-1 approach proved superior in reducing average blood glucose levels to a number of other dual combinations across 5 Phase III trials, including Forxiga plus metformin, Onglyza with metformin, or glimepiride with metformin.

Five drugs, in­clud­ing two No­var­tis ther­a­pies, win EMA en­dorse­ment

As is custom, an EMA panel on Friday issued its weekly recommendations on marketing applications submitted by drug developers. This week, the agency backed the use of five new therapies — including two Novartis drugs — but issued no negative reviews.

Novartis’ S1P drug for relapsing forms of multiple sclerosis (MS) drug, Mayzent (known chemically as siponimod), which was approved by the FDA in March — has been given the nod by the EMA. The Swiss drugmaker already sells its other MS drug, Gilenya, in both regions.

Atom­wise's X-37 spin­out gets $14.5 mil­lion to launch AI dis­cov­ery ef­forts

The folks behind Atomwise’s spinout X-37 like to think in cosmological metaphors, and you can think of their AI drug development model as probes sent into space from a central station. That station just got $14.5 million in Series A funding from DCVC Bio, Alpha Intelligence Capital and Hemi Ventures to back those missions.

X-37 uses Atomwise’s AI platform to identify drug targets and – unlike the parent company, which largely sticks to computers  – bring those into a wet lab and preclinical testing.  In addition to AI professionals, it’s led in by part by drug developers from Velocity Pharmaceutical Development.

Ab­bott Lab­o­ra­to­ries CEO Miles White pass­es ba­ton down to suc­ces­sor; Lon­za CEO Marc Funk hits the ex­it

→ Abbott Laboratories has named a successor to CEO Miles White after he announced that he was stepping down in March after 21 years of service. Robert Ford, the company’s COO and president, will take the helm. Ford is known for his work in the $25 billion merger between St. Jude Medical into Abbott in January 2017. White will remain with the company as executive chairman of the board. 

→ After snapping up Novartis’ Swiss facility, Novartis Center of Excellence, in July, Lonza has announced that their CEO, Marc Funk, is hitting the exit for “personal reasons.” Funk has been the CEO of the company for less than a year — brought onto the company back in March. In the meantime, chairman Albert Baehny will serve as interim CEO. 

BeiGene CEO John Oyler at an Endpoints event in Shanghai, October 2018 (Credit: Endpoints News/PharmCube)

UP­DAT­ED: In a first, FDA green-lights use of a Chi­nese built can­cer ther­a­py — and more are com­ing

Weeks after Amgen took a $2.7 billion stake in BeiGene, the Beijing-based biotech has secured its first-ever FDA approval for zanubrutinib, a BTK inhibitor, months ahead of schedule.

BeiGene’s drug, branded as Brukinsa, has secured accelerated approval for adult patients with mantle cell lymphoma (MCL) — a typically aggressive, rare, form of blood cancer — who have received at least one prior therapy.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,600+ biopharma pros reading Endpoints daily — and it's free.