AS­CO17: As­traZeneca pumps up ex­pec­ta­tions on near-term can­cer drug wins, look­ing to shed a rep for fail­ure

CHICA­GO — As­traZeneca’s R&D team ar­rived at AS­CO on a crit­i­cal mis­sion.

With its lead­ing PARP in­hibitor outscor­ing chemo in treat­ing breast can­cer,  mak­ing the spot­light round on Sun­day, As­traZeneca was able to of­fer some fresh, sol­id ev­i­dence of its con­tin­u­ing progress on the all-im­por­tant can­cer front. And with sev­er­al key cat­a­lysts lined up over the sum­mer, ex­ecs want­ed to send a clear mes­sage that this can­cer drug roll they’re on is just be­gin­ning.

As far as Leerink’s Sea­mus Fer­nan­dez is con­cerned, As­traZeneca’s in­vestor meet­ing in Chica­go was a rous­ing suc­cess, with some sug­ges­tions that the phar­ma gi­ant is on the verge of of­fer­ing some ‘prac­tice chang­ing’ ad­vances in the field.

Sean Bo­hen, As­traZeneca

As­traZeneca ex­ecs left the crowd with the clear im­pres­sion that Lyn­parza was on its way to a broad ap­proval for sec­ond-line main­te­nance ther­a­py in treat­ing ovar­i­an can­cer. R&D chief Sean Bo­hen al­so hint­ed that the full read­out on the PA­CIF­IC tri­al — Imfinzi monother­a­py fol­low­ing chemo and ra­di­a­tion in Stage 3 non-small cell lung can­cer — will be a hit. Fer­nan­dez not­ed:

Based on this and com­ments from pre­vi­ous­ly in­ter­viewed KOLs, we be­lieve the re­sults are like­ly to be both clin­i­cal­ly mean­ing­ful and prac­tice-chang­ing. AZN es­ti­mates a Stage III NSCLC in­ci­dence of ~100k per year, with about half be­ing un­re­sectable. With oth­er PD-1/L1 in­hibitors ~2-3 years be­hind in the set­ting, this rep­re­sents a large mar­ket-ex­pand­ing op­por­tu­ni­ty for Imfinzi.

Fer­nan­dez be­lieves that the phar­ma gi­ant has al­ready filed its BTK drug acal­abru­ti­nib for an ap­proval at the FDA and is just wait­ing for con­fir­ma­tion that the pack­age has been ac­cept­ed for re­view. Peak sales es­ti­mates for this drug scrape the $1 bil­lion mark— pos­si­bly mak­ing it one of the top 10 or­phan drugs in the late-stage pipeline — which would make it a valu­able ad­di­tion to the port­fo­lio.

And then there’s MYS­TIC, As­traZeneca’s com­bo of its check­point Imfinzi (dur­val­um­ab) and treme­li­mum­ab, a CT­LA-4 drug. Over the past few months there’s been a grow­ing sense that this com­bi­na­tion could well floun­der on CT­LA-4’s well-known tox­i­c­i­ty. But As­traZeneca’s team is keep­ing their smiles in place, try­ing to as­sure every­one that this pro­gram re­mains on track, with plen­ty of po­ten­tial to im­press.

The da­ta are ex­pect­ed in a mat­ter of weeks now, so this mys­tery won’t play long. A suc­cess here would make a huge dif­fer­ence for the multi­na­tion­al, which has been dogged by clin­i­cal set­backs for years now. Some as­sur­ance that CEO Pas­cal So­ri­ot’s big bet on can­cer is pay­ing off with a multi­bil­lion-dol­lar re­turn would go a long way to as­sur­ing an­a­lysts that the long-await­ed turn­around is near.

Fail­ure would like­ly blight the process once again.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Anthony Fauci (AP Images)

UP­DAT­ED: NIH-part­nered Mod­er­na ships off its PhI-ready coro­n­avirus vac­cine can­di­date to a sea of un­cer­tain­ty

Off it goes.

Moderna has shipped the first batch of its mRNA vaccine against SARS-CoV-2 from its manufacturing facility in Norwood, Massachusetts, to the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland, for a pioneering Phase I study.

It’s a hectic race against time. In the 42 days since Moderna selected the sequence they would use to develop their vaccine — a record time, no less — the number of confirmed cases around the world has surged astronomically from a few dozen to over 80,000, per WHO and Johns Hopkins estimates.

The candidate that they came up with, mRNA-1273, encodes for a prefusion stabilized form of the spike protein, which gives the virus its crown shape and plays a key role in transmission. The Coalition for Epidemic Preparedness Innovations, the Oslo-based group better known as CEPI, funded the manufacture of this batch.

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Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

Mallinck­rodt, once the na­tion’s largest oxy­codone pro­duc­er, an­nounces ten­ta­tive $1.6B set­tle­ment

Three years after it first paid out fines for its role in the US opioid abuse epidemic, Mallinckrodt has announced an agreement-in-principle that will see the company pay out $1.6 billion and place its generics unit in bankruptcy.

The tentative deal would settle hundreds of lawsuits from state and local governments over Mallinckrodt’s role in the epidemic, while also helping address the company’s increasingly mountainous debt. Although Purdue Pharma has drawn the bulk of both public and legal acrimony for opioid sales, documents made public earlier this year showed that Mallinckrodt subsidiary SpecGx, along with the generic subsidiaries of Teva and Endo Pharmaceuticals, accounted for the vast majority of the 76 billion opioid pills distributed from 2006 to 2012. Mallinckrodt was at the top of that list.

UP­DAT­ED: No­var­tis' plans to wres­tle Eylea mar­ket share take a hit as Beovu is linked to safe­ty con­cerns

While Regeneron’s flagship eye therapy Eylea hurtles towards a patent cliff, the sales of its main rival — Novartis’ Beovu — could be tainted by safety concerns.

On Sunday night, Chicago-based American Society of Retina Specialists (ASRS) issued a note to members about 14 cases of retinal vasculitis (including 11 cases of occlusive retinal vasculitis) — a sight-threatening inflammatory eye condition that involves the retinal vessels — across 46,000 injections administered since Beovu’s launch in November 2019, Wall Street analysts reported.

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UP­DAT­ED: NGM Bio takes leap for­ward in crowd­ed NASH field

South San Francisco-based NGM Bio may have underwhelmed with its interim analysis of a key cohort from a mid-stage NASH study last fall — but stellar topline data unveiled on Monday showed the compound induced significant signs of antifibrotic activity, NASH resolution and liver fat reduction, sending the company’s stock soaring.

There are an estimated 50+ companies focused on developing drugs for non-alcoholic steatohepatitis, or NASH, a common liver disease that has long flummoxed researchers. The first wave of NASH drug developers struggled with efficacy as well as safety — and companies big and small have crashed and burned.

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Mickey Kertesz, KidsandArtOrg via YouTube

Soft­Bank's newest, $165M biotech in­vest­ment looks for in­fec­tious traces in the blood

SoftBank has found its newest biotech investment.

The Japanese bank has invested $165 million into Karius, a company that uses blood tests to diagnose infectious diseases, as part of its new Vision Fund 2. The full scope of the new fund has yet to be announced, but the first and newly-beleaguered Vision Fund poured $100 billion into technology companies, including the biotechs Vir Biotechnology and Roivant and the sequencing company 10x Genomics.

Methicillin-resistant Staph aureus (Shutterstock)

FDA grants ‘break­through’ sta­tus to an­tibi­ot­ic al­ter­na­tive as Con­tra­Fect rush­es to join fight against su­per­bug

An experimental drug that promises to be the first anti-infective agent to prove superior to vancomycin — an antibiotic approved in 1958 — has notched the FDA’s “breakthrough” status.

ContraFect said the designation was based on Phase II data in which exebacase was tested against a superbug known as methicillin-resistant Staph aureus, or MRSA. In a subgroup analysis, the clinical responder rate at day 14 was 42.8% higher than that among those treated with standard of care, the company said (p=0.010).

Zhong Nanshan, CGTN via YouTube

Har­vard joins coro­n­avirus fight with $115M and a high-pro­file Chi­nese part­ner

For two months, as the novel coronavirus swelled from a few early cases tied to a Wuhan market to a global epidemic, most of the world’s focus and dollars have flowed toward emergency initiatives: building vaccines at a record pace, plucking experimental antivirals out of freezers to see what sticks and immunizing mice for new antibodies.

Now a new and well-funded collaboration between Harvard and a top Chinese research institute will play the long game. In a 5-year, $115 million initiative backed by China Evergrande Group, researchers from the Harvard Medical School, Harvard T.H. Chan School of Public Health and Guangzhou Institute for Respiratory Health will study the virus in an effort to develop therapies against infections by the novel coronavirus, known as SARS-CoV-2, and to prevent new ones.